90. 網膜色素変性症 Retinitis pigmentosa Clinical trials / Disease details
臨床試験数 : 147 / 薬物数 : 176 - (DrugBank : 43) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 110
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02018692 (ClinicalTrials.gov) | March 1, 2024 | 17/12/2013 | The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis Pigmentosa | The Effect of Oral Administration of 9-cis ß Carotene Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Dietary Supplement: Alga Dunaliella Bardawil powder;Dietary Supplement: Placebo:Starch | Sheba Medical Center | NULL | Not yet recruiting | 12 Years | 18 Years | All | 30 | Phase 1/Phase 2 | Israel |
| 2 | NCT05282953 (ClinicalTrials.gov) | November 10, 2022 | 8/3/2022 | A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa (ABACUS) | A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa (ABACUS) | Retinitis Pigmentosa | Drug: KIO-301 | Kiora Pharmaceuticals, Inc. | NULL | Recruiting | 18 Years | 80 Years | All | 6 | Phase 1 | Australia |
| 3 | NCT05413148 (ClinicalTrials.gov) | August 5, 2022 | 28/5/2022 | The Effect of Stem Cells and Stem Cell Exosomes on Visual Functions in Patients With Retinitis Pigmentosa | The Effect of Wharton Jelly-derived Mesenchymal Stem Cells and Stem Cell Exosomes on Visual Functions in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Biological: Subtenon injection of Wharton jelly derived mesenchymal stem cells;Biological: Subtenon injection of Wharton jelly derived mesenchymal stem cell exosomes;Other: Placebo | TC Erciyes University | NULL | Recruiting | 18 Years | 70 Years | All | 135 | Phase 2/Phase 3 | Turkey |
| 4 | EUCTR2020-002873-88-IE (EUCTR) | 19/07/2022 | 15/10/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: Botaretigene Sparoparvovec Product Code: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: Botaretigene Sparoparvovec Product Code: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | United States;Spain;Ireland;Israel;Switzerland;Italy;United Kingdom;France;Canada;Belgium;Denmark;Netherlands;Germany | ||
| 5 | NCT05392179 (ClinicalTrials.gov) | July 14, 2022 | 28/3/2022 | A Study in Subjects With Retinitis Pigmentosa | An Open-label, Phase II Study of ADX-2191 in Subjects With Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: ADX-2191 | Aldeyra Therapeutics, Inc. | NULL | Recruiting | 18 Years | N/A | All | 8 | Phase 2 | United States |
| 6 | NCT05392751 (ClinicalTrials.gov) | June 2, 2022 | 23/5/2022 | Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa | Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa | Retinitis Pigmentosa;Retinitis Pigmentosa Syndrome | Drug: EA-2353 | Endogena Therapeutics, Inc | NULL | Recruiting | 18 Years | N/A | All | 14 | Phase 1/Phase 2 | United States |
| 7 | EUCTR2020-002255-37-IE (EUCTR) | 26/05/2022 | 15/10/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | ||
| 8 | EUCTR2020-002255-37-ES (EUCTR) | 28/03/2022 | 02/11/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | ||
| 9 | EUCTR2020-002873-88-ES (EUCTR) | 28/03/2022 | 02/11/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | ||
| 10 | EUCTR2020-002873-88-NL (EUCTR) | 14/03/2022 | 22/10/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Netherlands;Germany | ||
| 11 | EUCTR2020-002255-37-NL (EUCTR) | 14/03/2022 | 22/10/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | Switzerland;United Kingdom;France;United States;Spain;Ireland;Israel;Italy;Canada;Belgium;Denmark;Netherlands;Germany | ||
| 12 | EUCTR2020-002873-88-DK (EUCTR) | 01/03/2022 | 08/11/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | ||
| 13 | EUCTR2020-002255-37-DK (EUCTR) | 01/03/2022 | 08/11/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | ||
| 14 | NCT05147701 (ClinicalTrials.gov) | February 1, 2022 | 29/11/2021 | Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for Eye Diseases | Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for the Treatment of Eye Diseases | Eye Diseases;Retinitis Pigmentosa;Glaucoma;Diabetic Retinopathy;Macular Degeneration;Traumatic Optic Neuropathy;Optic Atrophy | Biological: AlloRx | The Foundation for Orthopaedics and Regenerative Medicine | NULL | Recruiting | N/A | N/A | All | 20 | Phase 1 | Antigua and Barbuda;Argentina;Mexico |
| 15 | NCT05203939 (ClinicalTrials.gov) | January 24, 2022 | 16/11/2021 | The Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis | Phase 1/2 Study to Assess the Safety and Efficacy of Ocu400 for Retinitis Pigmentosa Associated With Nr2e3 and Rho Mutations and Leber Congenital Amaurosis With Mutation(S) in Cep290 Gene | Retinitis Pigmentosa;Leber Congenital Amaurosis | Drug: OCU400 Low Dose;Drug: OCU400 Mid Dose;Drug: OCU400 High Dose | Ocugen | NULL | Recruiting | 18 Years | N/A | All | 21 | Phase 1/Phase 2 | United States |
| 16 | NCT05176717 (ClinicalTrials.gov) | December 15, 2021 | 23/11/2021 | Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Active, not recruiting | 12 Years | N/A | All | 5 | Phase 2/Phase 3 | United States;United Kingdom |
| 17 | NCT05158296 (ClinicalTrials.gov) | December 8, 2021 | 23/11/2021 | Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius) | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius) | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: Ultevursen;Other: Sham-procedure | ProQR Therapeutics | NULL | Active, not recruiting | 12 Years | N/A | All | 81 | Phase 2/Phase 3 | United States;Germany;Netherlands;United Kingdom |
| 18 | NCT05085964 (ClinicalTrials.gov) | September 16, 2021 | 7/10/2021 | An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa | An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia) | Retinitis Pigmentosa;Usher Syndrome Type 2 | Drug: RNA antisense oligonucleotide for intravitreal injection | ProQR Therapeutics | NULL | Terminated | 12 Years | N/A | All | 21 | Phase 2 | United States;Canada;France |
| 19 | NCT04850118 (ClinicalTrials.gov) | August 2021 | 5/4/2021 | A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations | A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene | X-Linked Retinitis Pigmentosa | Biological: rAAV2tYF-GRK1-hRPGRco | Applied Genetic Technologies Corp | NULL | Not yet recruiting | 8 Years | 50 Years | Male | 63 | Phase 2/Phase 3 | Israel;Netherlands;United States |
| 20 | NCT04864496 (ClinicalTrials.gov) | April 17, 2021 | 21/4/2021 | Effects of Treatment With N- Acetylcysteine on Visual Outcomes in Patients With Retinitis Pigmentosa | Effects of Oral N- Acetylcysteine on Macular Function in Retinitis Pigmentosa; a Phase 2 Randomized Controlled Trial | Retinitis Pigmentosa | Drug: Prescribe N-acetylcysteine tablets;Drug: Prescribe placebo tablets | Shahid Beheshti University of Medical Sciences | NULL | Active, not recruiting | 18 Years | 40 Years | All | 30 | Phase 2 | Iran, Islamic Republic of |
| 21 | NCT04658251 (ClinicalTrials.gov) | March 3, 2021 | 1/12/2020 | Study of New Mutations in Cone Disorders | Functional Study of Intronic Variants in Inherited Cone Disorders | Retinal Dystrophy, Cone-Rod;Cone Dystrophy;Cone Rod Dystrophy;Macular Degeneration | Genetic: Blood and/or skin biopsy | University Hospital, Lille | NULL | Recruiting | 3 Years | N/A | All | 20 | France | |
| 22 | EUCTR2017-003104-42-DK (EUCTR) | 08/02/2021 | 01/07/2020 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2- REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 Product Code: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR INN or Proposed INN: Cotoretigene Toliparvovec Other descriptive name: AAV8-RPGR | NightstaRx Ltd (A Biogen Company) | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | Netherlands;Germany;United Kingdom;France;United States;Canada;Finland;Brazil;Denmark | ||
| 23 | NCT04636853 (ClinicalTrials.gov) | December 23, 2020 | 14/11/2020 | CB-PRP in Retinitis Pigmentosa and Dry Age-related Macular Degeneration | Cord Blood Platelet-rich Plasma (CB-PRP) in Retinitis Pigmentosa and Dry Age-related Macular Degeneration | Retinitis Pigmentosa;Dry Age-related Macular Degeneration | Biological: Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | NULL | Completed | 18 Years | 70 Years | All | 20 | Phase 3 | Italy |
| 24 | EUCTR2019-004547-77-GB (EUCTR) | 09/06/2020 | 26/02/2020 | Safety and Tolerability of hRPC in Retinitis Pigmentosa | First-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Subjects with Retinitis Pigmentosa (RP) | Retinitis Pigmentosa MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: human Retinal Progenitor Cells Drug Product Product Code: hRPC DP INN or Proposed INN: ALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED Other descriptive name: ALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED | ReNeuron Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 2 | United States;United Kingdom | ||
| 25 | NCT04517149 (ClinicalTrials.gov) | June 9, 2020 | 14/8/2020 | 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene | X-Linked Retinitis Pigmentosa | Biological: 4D-125 IVT Injection;Other: Observational | 4D Molecular Therapeutics | NULL | Active, not recruiting | 12 Years | N/A | Male | 21 | Phase 1/Phase 2 | United States |
| 26 | NCT04278131 (ClinicalTrials.gov) | February 6, 2020 | 13/2/2020 | BS01 in Patients With Retinitis Pigmentosa | Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: BS01 | Bionic Sight LLC | NULL | Recruiting | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States |
| 27 | NCT04919473 (ClinicalTrials.gov) | October 23, 2019 | 25/5/2021 | Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa | A Phase I/IIa Open Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa | Retinitis Pigmentosa;Retinal Diseases;Retinal Degeneration | Biological: Gene Therapy product:vMCO-I | Nanoscope Therapeutics Inc. | NULL | Completed | 18 Years | N/A | All | 11 | Phase 1/Phase 2 | India |
| 28 | NCT05712148 (ClinicalTrials.gov) | October 11, 2019 | 16/1/2023 | Spheroidal Mesenchymal Stem Cells in Retinitis Pigmentosa | Suprachoroidal Spheroidal Mesenchymal Stem Cell Implantation in Retinitis Pigmentosa: Clinical Results of 6 Months Follow-up | Retinitis Pigmentosa;Stem Cell Transplant | Biological: Stem cell implantation | Acibadem University | NULL | Completed | N/A | N/A | All | 15 | Phase 1/Phase 2 | Turkey |
| 29 | NCT04123626 (ClinicalTrials.gov) | October 7, 2019 | 1/10/2019 | A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene | A Prospective First-In-Human Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) Due to the P23H Mutation in the RHO Gene | Autosomal Dominant Retinitis Pigmentosa;Eye Diseases;Eye Diseases, Hereditary;Retinal Dystrophies;Retinal Disease;Retinitis;Vision Tunnel;Vision Disorders | Drug: QR-1123;Other: Sham procedure | ProQR Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 11 | Phase 1/Phase 2 | United States |
| 30 | NCT04611503 (ClinicalTrials.gov) | September 24, 2019 | 20/5/2020 | PDE6A Gene Therapy for Retinitis Pigmentosa | PIGMENT - PDE6A Gene Therapy for Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: subretinal injection of rAAV.hPDE6A | STZ eyetrial | NULL | Active, not recruiting | 18 Years | N/A | All | 9 | Phase 1/Phase 2 | Germany |
| 31 | NCT03963154 (ClinicalTrials.gov) | August 19, 2019 | 22/5/2019 | Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation | STREAM: A Phase 1/2, Open-label, Safety, Tolerability and Preliminary Efficacy Study of Implantation Into One Eye of hESC-derived RPE in Patients With Retinitis Pigmentosa Due to Monogenic Mutation | Retinitis Pigmentosa | Biological: Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE) | Centre d'Etude des Cellules Souches | NULL | Active, not recruiting | 18 Years | 65 Years | All | 7 | Phase 1/Phase 2 | France |
| 32 | EUCTR2016-003705-34-DE (EUCTR) | 02/07/2019 | 26/11/2018 | Administration of a genetically modified virus particle in patients with PDE6A-linked retinitis pigmentosa | PIGMENT – PDE6A gene therapy for retinitis pigmentosa | PDE6A-linked retinitis pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: rAAV.hPDE6A vector | Universitätsklinikum Tübingen | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Germany | ||
| 33 | NCT03999021 (ClinicalTrials.gov) | June 24, 2019 | 24/6/2019 | FIGHT-RP 1 Extension Study | A Phase 1 Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of N-Acetylcysteine (NAC) in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: NAC effervescent tablets | Johns Hopkins University | NULL | Active, not recruiting | 18 Years | N/A | All | 30 | Phase 1 | United States |
| 34 | NCT04224207 (ClinicalTrials.gov) | April 1, 2019 | 6/1/2020 | Management of Retinitis Pigmentosa by Mesenchymal Stem Cells by Wharton's Jelly Derived Mesenchymal Stem Cells | Management of Retinitis Pigmentosa by Wharton's Jelly Derived Mesenchymal Stem Cells: Preliminary Clinical Results | Retinitis Pigmentosa;Inherited Retinal Dystrophy | Biological: Wharton's jelly derived mesenchymal stem cell | Ankara Universitesi Teknokent | NULL | Completed | 18 Years | 60 Years | All | 32 | Phase 3 | Turkey |
| 35 | NCT03780257 (ClinicalTrials.gov) | March 6, 2019 | 17/12/2018 | Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene | A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only) | ProQR Therapeutics | NULL | Completed | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States;Canada;France;Belgium |
| 36 | NCT04068207 (ClinicalTrials.gov) | February 22, 2019 | 23/8/2019 | Minocycline Treatment in Retinitis Pigmentosa | The Efficacy and Safety of Oral Minocycline in the Treatment of Retinitis Pigmentosa: An Open-label Clinical Trial | Retinitis Pigmentosa;Inherited Retinal Dystrophy;Retina Disorder | Drug: Minocycline | Sun Yat-sen University | NULL | Recruiting | 18 Years | 60 Years | All | 35 | Phase 2 | China |
| 37 | NCT04312672 (ClinicalTrials.gov) | February 18, 2019 | 15/7/2019 | Long Term Follow-Up Gene Therapy Study for XLRP RPGR | Long Term Follow-Up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-Associated Virus Vector (AAV2-RPGR) for Gene Therapy of Adults and Children With X-Linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR) | X-Linked Retinitis Pigmentosa | Biological: AAV-RPGR | MeiraGTx UK II Ltd | NULL | Recruiting | 5 Years | N/A | Male | 36 | Phase 1/2 | United States;United Kingdom |
| 38 | EUCTR2018-002433-38-FR (EUCTR) | 15/02/2019 | 16/11/2018 | Study to evaluate QR-421a in subjects with retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A Gene | A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene - STELLAR | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Canada;Belgium | ||
| 39 | EUCTR2013-005393-22-NL (EUCTR) | 04/01/2019 | 20/09/2016 | A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision. | A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) | Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QLT091001 Product Code: QLT091001 INN or Proposed INN: Zuretinol Acetate Other descriptive name: QLT091001 | QLT Inc. | NULL | NA | Female: yes Male: yes | 48 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Brazil;Denmark;Germany;Netherlands;United Kingdom;Switzerland | ||
| 40 | NCT03772938 (ClinicalTrials.gov) | December 13, 2018 | 3/11/2018 | Stem Cells Therapy in Degenerative Diseases of the Retina | Stem Cells Therapy in Degenerative Diseases of the Retina | Retinal Degeneration;Retinitis Pigmentosa;Age Related Macular Degeneration;Stargardt Disease 1 | Biological: Stem/progenitor cells transplantation | Pomeranian Medical University Szczecin | NULL | Unknown status | 18 Years | 65 Years | All | 30 | Phase 1 | Poland |
| 41 | EUCTR2017-003104-42-FI (EUCTR) | 20/11/2018 | 16/10/2018 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 Product Code: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR INN or Proposed INN: Cotoretigene Toliparvovec Other descriptive name: AAV8-RPGR | NightstaRx Ltd (A Biogen Company) | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | France;United States;Canada;Finland;Brazil;Denmark;Netherlands;Germany;United Kingdom | ||
| 42 | NCT04315025 (ClinicalTrials.gov) | October 7, 2018 | 8/3/2020 | Safety Issues of Peribulbar Injection of UC-MSC in Patients With Retinitis Pigmentosa | Safety Issues of Peribulbar Injection of Umbilical Cord Mesenchymal Stem Cell (UC-MSC) in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Biological: Umbilical Cord Mesenchymal Stem Cell (UC-MSC);Biological: Conditioned Medium (CM) | PT. Prodia Stem Cell Indonesia | NULL | Completed | 18 Years | 65 Years | All | 18 | Phase 1/Phase 2 | Indonesia |
| 43 | EUCTR2018-000425-31-GB (EUCTR) | 30/08/2018 | 14/06/2018 | Long term follow-up study of gene therapy trial for X-linked Retinitis Pigmentosa | Long term follow-up study of participants following an open label, multicentre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Long term follow-up gene therapy study for X-linked Retinitis Pigmentosa | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2/5-hRKp.RPGR | MeiraGTx UK II Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 36 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 44 | NCT03374657 (ClinicalTrials.gov) | August 22, 2018 | 11/12/2017 | A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa | An Open-label First-in-human Single Ascending Dose Study to Explore Safety, Tolerability and Efficacy of Subretinal Administration of CPK850 Gene Therapy in Patients With Retinitis Pigmentosa Due to Mutations in the Retinaldehyde Binding Protein 1 (RLBP1) Gene | Retinitis Pigmentosa | Biological: CPK850 | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 70 Years | All | 12 | Phase 1/Phase 2 | Sweden |
| 45 | EUCTR2017-003104-42-GB (EUCTR) | 26/06/2018 | 06/02/2018 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study - SOLSTICE | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 Product Code: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: AAV2-REP1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR Other descriptive name: AAV8-RPGR | NightstaRx Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom | ||
| 46 | NCT03584165 (ClinicalTrials.gov) | June 4, 2018 | 29/6/2018 | Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia;X-Linked Retinitis Pigmentosa | Genetic: BIIB111;Genetic: BIIB112 | NightstaRx Ltd, a Biogen Company | NULL | Enrolling by invitation | 10 Years | N/A | Male | 440 | Phase 3 | United States;Brazil;Canada;Denmark;Finland;France;Germany;Netherlands;United Kingdom |
| 47 | EUCTR2016-002696-10-SE (EUCTR) | 09/05/2018 | 21/12/2017 | Explore the safety and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene | An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene | retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: CPK850 INN or Proposed INN: TBD | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 21 | Phase 1;Phase 2 | Sweden | ||
| 48 | NCT03316560 (ClinicalTrials.gov) | April 16, 2018 | 10/10/2017 | Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations | A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene | X-Linked Retinitis Pigmentosa | Biological: rAAV2tYF-GRK1-RPGR | Applied Genetic Technologies Corp | NULL | Recruiting | 6 Years | 50 Years | Male | 42 | Phase 1/Phase 2 | United States |
| 49 | EUCTR2017-001139-38-PT (EUCTR) | 12/02/2018 | 15/11/2017 | MK-3475 (SCH 9000475) as neoadjuvant and adjuvant therapy in Stage III-IVA resectable LA HNSCC (Locoregionally Advanced Head and Neck Squamous Cell Carcinoma) | A Phase III, Randomized, Open-label Study to Evaluate Pembrolizumab as Neoadjuvant Therapy and in Combination With Standard of Care as Adjuvant Therapy for StageIII-IVA Resectable Locoregionally Advanced Head and Neck Squamous Cell Carcinoma (LA HNSCC) - MK-3475 (SCH 9000475) as neoadjuvant and adjuvant therapy in Stage III-IVA resectable LA HNSCC | Retinitis Pigmentosa, associated with Ushers Syndrome Type 1B. MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10063396;Term: Usher's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: UshStat (SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed INN: Lentiviral vector containing the human MY07A gene Other descriptive name: UshStat | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc. | NULL | Not Recruiting | Female: yes Male: yes | 704 | Phase 1;Phase 2 | United States;France | ||
| 50 | EUCTR2017-003104-42-DE (EUCTR) | 05/01/2018 | 25/09/2017 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RetinalGene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR Other descriptive name: AAV8-RPGR | NightstaRx Ltd (A Biogen Company) | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom | ||
| 51 | EUCTR2017-002204-27-GB (EUCTR) | 22/12/2017 | 20/10/2017 | A Phase 1/2a gene therapy clinical trial in Retinitis Pigmentosa subjects | A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa | Retinitis Pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: Recombinant adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene Product Code: GS030-DP | GENSIGHT-BIOLOGICS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 52 | NCT03328130 (ClinicalTrials.gov) | November 6, 2017 | 5/10/2017 | Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene | Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression | Retinitis Pigmentosa | Biological: AAV2/5-hPDE6B | Coave Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 17 | Phase 1/Phase 2 | France |
| 53 | EUCTR2016-001429-16-FR (EUCTR) | 28/07/2017 | 12/09/2017 | Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients Harboring Mutations in the PDE6B Gene Leading to a Defect in PDE6B Expression. | Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients Harboring Mutations in the PDE6B Gene Leading to a Defect in PDE6B Expression. | Retinitis Pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: HORA-PDE6B (AAV2/5.hPDE6B) | HORAMA SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 1;Phase 2 | France | |||
| 54 | EUCTR2016-003967-21-GB (EUCTR) | 09/06/2017 | 14/09/2017 | Gene Therapy Trial for People with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Gene Therapy Trial for People with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2/5-hRKp.RPGR INN or Proposed INN: AAV2/5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 71 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 55 | NCT03116113 (ClinicalTrials.gov) | March 16, 2017 | 29/3/2017 | A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 | A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) | X-Linked Retinitis Pigmentosa | Biological: BIIB112 | NightstaRx Ltd, a Biogen Company | NULL | Completed | 10 Years | N/A | Male | 50 | Phase 1/Phase 2 | United States;United Kingdom |
| 56 | NCT03063021 (ClinicalTrials.gov) | February 15, 2017 | 16/2/2017 | The FIGHT-RP1 Study | A Phase 1 Open Label Dose Ranging Study to Assess the Safety and Tolerability of N-Acetylcysteine (NAC) in Patients With Retinitis Pigmentosa (FIGHT-RP1 Study) | Retinitis Pigmentosa | Drug: N-Acetyl Cysteine (NAC) | Johns Hopkins University | NULL | Completed | 18 Years | N/A | All | 30 | Phase 1 | United States |
| 57 | EUCTR2016-003852-60-GB (EUCTR) | 09/01/2017 | 09/11/2016 | A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa using AAV8 | A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) - XIRIUS Study | X-Linked retinitis pigmentosa (XLRP) MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV8-RPGR Product Code: AAV8-RPGR Other descriptive name: AAV8-RPGR | NightstaRx Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 63 | Phase 1;Phase 2;Phase 3 | United States;United Kingdom | ||
| 58 | NCT04252534 (ClinicalTrials.gov) | January 1, 2017 | 31/1/2020 | Management of Retinitis Pigmentosa Via Electromagnetic Stimulation and Platelet Rich Plasma | Management of Retinitis Pigmentosa Via Repetitive Electromagnetic Stimulation and Autologous Platelet Rich Plasma | Retinitis Pigmentosa | Combination Product: PRP combined Magnovision;Biological: PRP | Ankara Universitesi Teknokent | NULL | Completed | 18 Years | 60 Years | All | 60 | N/A | NULL |
| 59 | EUCTR2013-005393-22-DK (EUCTR) | 03/11/2016 | 06/09/2016 | A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision. | A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) | Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QLT091001 Product Code: QLT091001 INN or Proposed INN: Zuretinol Acetate Other descriptive name: QLT091001 | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 3 | United States;France;Canada;Brazil;Denmark;Netherlands;Germany;United Kingdom;Switzerland | ||
| 60 | EUCTR2016-002523-28-GB (EUCTR) | 16/09/2016 | 05/07/2016 | A Pilot Study Evaluating the Effect of Intravitreal Fluocinolone Acetonide (0.19mg) in Patients with Retinitis Pigmentosa. | A Pilot Study Evaluating the Effect of Intravitreal Fluocinolone Acetonide (0.19mg) in Patients with Retinitis Pigmentosa - Iluvien for IRD | Retinitis Pigmetosa. MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Iluvien Product Name: Iluvien Product Code: n/a INN or Proposed INN: Fluocinolone Acetonide | Moorfields Eye Hospital | NULL | Not Recruiting | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 61 | JPRN-UMIN000023618 | 2016/09/01 | 01/09/2016 | Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa | Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa - Posterior capsule opacification in patients with retinitis pigmentosa | Cataract associated with patients with retinitis pigmentosa | Cataract surgery and implantation of 1P intraocular lens (PCB00V) Cataract surgery and implantation of 3P intraocular lens (VA-70AD) | Kyushu University Hospital | NULL | Complete: follow-up complete | 40years-old | Not applicable | Male and Female | 40 | Not selected | Japan |
| 62 | NCT04238858 (ClinicalTrials.gov) | September 1, 2016 | 18/1/2020 | Effects of Subtenon-injected Autologous Platelet-rich Plasma on Visual Functions in Eyes With Retinitis Pigmentosa | Effects of Subtenon-injected Autologous Platelet-rich Plasma on Visual Functions in Eyes With Retinitis Pigmentosa | Retinitis Pigmentosa | Biological: Platelet rich plasma | Ankara Universitesi Teknokent | NULL | Completed | 18 Years | 55 Years | All | 48 | N/A | Turkey |
| 63 | JPRN-UMIN000022686 | 2016/07/01 | 01/07/2016 | The Clinical Efficacy of Carbonic Anhydrase Inhibitors and Steroids in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa | The Clinical Efficacy of Carbonic Anhydrase Inhibitors and Steroids in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa - The Clinical Efficacy in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa | retinitis pigmentosa | 1% dorzolamide eye drop three times daily in each eye 0.1% betamethasone sodium phosphate eye drop three times daily in each eye oral 250mg acetazolamide two tablets daily 40mg triamcinolone acetonide into the sub-Tenon capsule | Kyushu University Hospital | NULL | Complete: follow-up complete | 20years-old | Not applicable | Male and Female | 40 | Not applicable | Japan |
| 64 | NCT02837640 (ClinicalTrials.gov) | June 2016 | 22/6/2016 | Studying a Potential Protective Effect of L-Dopa on Retinitis Pigmentosa | The Effect of L-Dopa on the Progression of Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: levodopa-carbidopa | Beirut Eye Specialist Hospital | NULL | Recruiting | 10 Years | 65 Years | Both | 50 | Phase 2 | Lebanon |
| 65 | EUCTR2015-003723-65-GB (EUCTR) | 12/01/2016 | 06/01/2016 | Prospective non-randomised pilot study looking at the safety and efficacy of a drug called Eylea for patients with an eye condition called Retinitis Pigmentosa, that also have swelling at the back of the eye | Aflibercept (Eylea®) for macular oedema associated with underlying Retinitis Pigmentosa (AMOUR) - AMOUR | Retinitis Pigmentosa associated Cystoid Macular Oedema MedDRA version: 18.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Trade Name: Eylea Product Name: Eylea Product Code: n/a INN or Proposed INN: Aflibercept | Moorfields Eye Hospital | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 66 | NCT02556736 (ClinicalTrials.gov) | December 14, 2015 | 21/9/2015 | RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa | Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP) | Advanced Retinitis Pigmentosa | Drug: RST-001 | AbbVie | NULL | Active, not recruiting | 18 Years | N/A | All | 14 | Phase 1/Phase 2 | United States |
| 67 | NCT02464436 (ClinicalTrials.gov) | December 2015 | 18/5/2015 | Safety and Tolerability of hRPC in Retinitis Pigmentosa | First-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Patients With Retinitis Pigmentosa (RP) | Retinitis Pigmentosa | Drug: hRPC | ReNeuron Limited | NULL | Active, not recruiting | 18 Years | N/A | All | 29 | Phase 1/Phase 2 | United States;Spain;United Kingdom |
| 68 | NCT02309866 (ClinicalTrials.gov) | January 2015 | 24/11/2014 | Identifying Genes and Mutations Underlying Retinitis Pigmentosa and Allied Diseases | Retinitis Pigmentosa | Genetic: Genetic testing | Hillel Yaffe Medical Center | Technion, Israel Institute of Technology | Not yet recruiting | N/A | N/A | Both | 100 | N/A | NULL | |
| 69 | NCT04356716 (ClinicalTrials.gov) | November 11, 2014 | 20/4/2020 | Sildenafil for Treatment of Choroidal Ischemia | Sildenafil for Treatment of Choroidal Ischemia | Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis Pigmentosa | Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA) | Columbia University | NULL | Recruiting | 18 Years | N/A | All | 25 | Phase 2 | United States |
| 70 | EUCTR2014-000385-22-IT (EUCTR) | 07/10/2014 | 12/05/2014 | Nerve Growth Factor eye drops as a novel treatment for patients with Retinitis Pigmentosa. | Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical toclinical Phase II trial. - NEMO | Retinitis pigmentosa with cystoid macular edema MedDRA version: 17.0;Level: LLT;Classification code 10054467;Term: Macular edema;System Organ Class: 100000004853 MedDRA version: 17.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: recombinant human Nerve Growth Factor (rhNGF) INN or Proposed INN: not yet assigned Other descriptive name: RECOMBINANT HUMAN NERVE GROWTH FACTOR (RHNGF) | Ospedale San Raffaele di Milano | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Italy | |||
| 71 | NCT01914913 (ClinicalTrials.gov) | September 2014 | 31/7/2013 | Clinical Study to Evaluate Safety and Efficacy of BMMNC in Retinitis Pigmentosa | An Open Labeled Clinical Study to Evaluate the Safety and Efficacy OF Autologous Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) in Retinitis Pigmentosa. It is Self Funded (Patients' Own Funding) Clinical Trial | Retinitis Pigmentosa | Biological: BMMNCs | Chaitanya Hospital, Pune | NULL | Recruiting | 18 Years | 65 Years | Both | 15 | Phase 1/Phase 2 | India |
| 72 | NCT02709876 (ClinicalTrials.gov) | April 2014 | 26/2/2016 | Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa | Retinitis Pigmentosa | Biological: Stem Cell Transplantation | Stem Cells Arabia | NULL | Active, not recruiting | 18 Years | 55 Years | All | 50 | Phase 1/Phase 2 | Jordan | |
| 73 | NCT02110225 (ClinicalTrials.gov) | January 2014 | 8/4/2014 | A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP) | A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP. | Retinitis Pigmentosa | Drug: rhNGF 60 µg/ml eye drops solution;Drug: rhNGF 180 µg/ml eye drops solution;Drug: Placebo | Dompé Farmaceutici S.p.A | NULL | Completed | 18 Years | N/A | All | 50 | Phase 1/Phase 2 | Italy |
| 74 | EUCTR2012-002574-31-FR (EUCTR) | 25/10/2013 | 15/05/2014 | Study of SAR421869 in Patients With Retinitis Pigmentosa associated with Usher Syndrome Type 1B | A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B | Retinitis Pigmentosa, associated with Ushers Syndrome Type 1B. MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10063396;Term: Usher's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: UshStat (SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed INN: Lentiviral vector containing the human MY07A gene Other descriptive name: UshStat | sanofi-aventis recherche & développement | NULL | Not Recruiting | Female: yes Male: yes | 27 | Phase 1;Phase 2 | United States;France | ||
| 75 | NCT01786395 (ClinicalTrials.gov) | March 2013 | 23/1/2013 | Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa | Phase III Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration Period | Retinitis Pigmentosa | Drug: UF-021;Drug: Placebo | R-Tech Ueno, Ltd. | NULL | Terminated | 20 Years | 70 Years | Both | 202 | Phase 3 | Japan |
| 76 | EUCTR2012-002436-82-ES (EUCTR) | 13/12/2012 | 22/10/2012 | Melatonin effects on visual and sleep patterns anomalies in subjects diagnosed with Retinitis Pigmentosa | Melatonin effects on visual and sleep patterns anomalies in subjects diagnosed with Retinitis Pigmentosa | To evaluate the sleep quality in patients with Retinitis Pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Trade Name: CIRCADIN 2 mg comprimidos de liberación prolongada Product Name: Circadin 2 mg INN or Proposed INN: Not answered Other descriptive name: MELATONIN | Isabel Pinilla Lozano | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Spain | ||||
| 77 | EUCTR2011-004214-42-NL (EUCTR) | 04/10/2012 | 23/05/2012 | A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | Leber Congenital AmaurosisRetinitis pigmentosa MedDRA version: 14.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: QLT091001 | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 31 | United States;Canada;Germany;Netherlands;United Kingdom | |||
| 78 | EUCTR2011-004214-42-GB (EUCTR) | 03/09/2012 | 02/05/2012 | A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | Leber Congenital Amaurosis Retinitis pigmentosa MedDRA version: 20.0;Level: PT;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: QLT091001 | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 31 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Netherlands;Germany;United Kingdom | ||
| 79 | NCT01680510 (ClinicalTrials.gov) | September 2012 | 4/9/2012 | The Effect of Oral Administration of 9-cis ß Carotene Rich Powder of the Alga Dunaliella Bardawil | The Effect of Oral Administration of 9-cis ß Carotene Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Dietary Supplement: Alga Dunaliella Bardawil;Other: Placebo (starch) | Sheba Medical Center | NULL | Active, not recruiting | 18 Years | N/A | All | 100 | Phase 1/Phase 2 | Israel |
| 80 | EUCTR2011-004214-42-DE (EUCTR) | 06/08/2012 | 03/05/2012 | A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | Leber Congenital AmaurosisRetinitis pigmentosa MedDRA version: 14.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: QLT091001 | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 31 | United States;Canada;Netherlands;Germany;United Kingdom | |||
| 81 | NCT01505062 (ClinicalTrials.gov) | March 26, 2012 | 4/1/2012 | Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B | A Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B | Usher Syndrome;Retinitis Pigmentosa | Drug: SAR421869 | Sanofi | NULL | Terminated | 6 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;France |
| 82 | NCT01543906 (ClinicalTrials.gov) | February 2012 | 17/2/2012 | Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) | An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) | Retinitis Pigmentosa (RP) | Drug: QLT091001 | QLT Inc. | NULL | Completed | 18 Years | 70 Years | Both | 5 | Phase 1 | Canada;Ireland |
| 83 | NCT01521793 (ClinicalTrials.gov) | January 2012 | 6/1/2012 | Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | LCA (Leber Congenital Amaurosis);RP (Retinitis Pigmentosa) | Drug: QLT091001 | QLT Inc. | NULL | Completed | 5 Years | 65 Years | Both | 27 | Phase 1 | United States;Canada;Germany;Netherlands;United Kingdom |
| 84 | NCT01560715 (ClinicalTrials.gov) | June 2011 | 19/3/2012 | Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa | Phase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa | Retinitis Pigmentosa | Biological: intravitreal injection of autologous bone marrow stem cells | University of Sao Paulo | NULL | Completed | 18 Years | 75 Years | All | 50 | Phase 2 | Brazil |
| 85 | NCT01399515 (ClinicalTrials.gov) | March 2011 | 3/5/2011 | Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa | Retinitis Pigmentosa;Retinal Diseases;Eye Diseases;Eye Disease, Hereditary;Retinal Degeneration | Drug: Valproic Acid | Seoul National University Hospital | NULL | Completed | 18 Years | N/A | Both | 200 | Phase 2 | Korea, Republic of | |
| 86 | JPRN-UMIN000005852 | 2010/06/01 | 27/06/2011 | The Clinical Efficacy of a Topical Dorzolamide in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa | The Clinical Efficacy of a Topical Dorzolamide in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa - The Clinical Efficacy of a Topical Dorzolamide in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa | retinitis pigmentosa | 1% dorzolamide three times daily in each eye during the follow-up period | Kyushu university hospital | NULL | Complete: follow-up complete | 20years-old | Not applicable | Male and Female | 20 | Not applicable | Japan |
| 87 | EUCTR2010-019079-32-PT (EUCTR) | 19/05/2010 | 05/04/2010 | A Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery System | A Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery System | Patients who participated in a previous intravitreal Brimo PS DDS study, including 190342-028D (Retinitis pigmentosa) and 190342-032D (Geographic Atrophy from Age-related Macular Degeneration). | Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System Product Code: 9715X INN or Proposed INN: Brimonidine Tartrate Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System Product Code: 9742X INN or Proposed INN: Brimonidine Tartrate Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System Product Code: 9741X INN or Proposed INN: Brimonidine Tartrate | Allergan Limited | NULL | Not Recruiting | Female: yes Male: yes | 290 | Portugal;Czech Republic;Germany;Italy;United Kingdom | |||
| 88 | EUCTR2010-019079-32-DE (EUCTR) | 12/05/2010 | 13/04/2010 | A Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery System | A Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery System | Patients who participated in a previous intravitreal Brimo PS DDS study, including 190342-028D (Retinitis pigmentosa) and 190342-032D (Geographic Atrophy from Age-related Macular Degeneration). | Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System Product Code: 9715X INN or Proposed INN: Brimonidine Tartrate Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System Product Code: 9742X INN or Proposed INN: Brimonidine Tartrate Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System Product Code: 9741X INN or Proposed INN: Brimonidine Tartrate | Allergan Limited | NULL | Not Recruiting | Female: yes Male: yes | 290 | Portugal;Czech Republic;Germany;Italy;United Kingdom | |||
| 89 | NCT01014052 (ClinicalTrials.gov) | November 2009 | 12/11/2009 | Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations | Phase 1b Study to Evaluate QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) | LCA (Leber Congenital Amaurosis);RP (Retinitis Pigmentosa) | Drug: QLT091001 | QLT Inc. | NULL | Completed | 5 Years | 65 Years | Both | 32 | Phase 1 | United States;Canada;Germany;Netherlands;United Kingdom |
| 90 | EUCTR2008-004561-26-IT (EUCTR) | 01/06/2009 | 11/11/2008 | Inner Retinal Dysfunction of the Cone System in Inherited Photoreceptor Degenerations: A Study of Disease Sequence and Assessment of Novel Therapeutic Strategies. - ND | Inner Retinal Dysfunction of the Cone System in Inherited Photoreceptor Degenerations: A Study of Disease Sequence and Assessment of Novel Therapeutic Strategies. - ND | retinitis pigmentosa MedDRA version: 9.1;Level: LLT;Classification code 10038914;Term: Retinitis pigmentosa MedDRA version: 9.1;Level: PT;Term: Retinitis pigmentosa | Product Name: Nerve Growt Factor - 2.5S INN or Proposed INN: COQUN | POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Italy | ||||
| 91 | NCT01068561 (ClinicalTrials.gov) | May 2009 | 10/2/2010 | Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa | Phase 1 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa | Retinitis Pigmentosa | Biological: intravitreal injection of autologous bone marrow stem cells | University of Sao Paulo | NULL | Completed | 18 Years | N/A | Both | 5 | Phase 1 | Brazil |
| 92 | JPRN-UMIN000006820 | 2009/02/01 | 02/12/2011 | Neuroprotective efficacy of topical unoprostone isopropyl or nipradilol in patients with retinitis pigmentosa | Neuroprotective efficacy of topical unoprostone isopropyl or nipradilol in patients with retinitis pigmentosa - Neuroprotective efficacy of topical unoprostone isopropyl or nipradilol in patients with retinitis pigmentosa | retinitis pigmentosa | 0.12% topical unoprostone isopropyl twice daily 0.25% topical nipradilol twice daily | Kyushu university hospital | NULL | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 50 | Not selected | Japan |
| 93 | NCT01773278 (ClinicalTrials.gov) | December 2008 | 3/12/2012 | Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS) | Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS) | Smith-Lemli-Opitz Syndrome;Cone-Rod Dystrophy;Hearing Loss | Drug: Antioxidants;Drug: Cholesterol | University of Colorado, Denver | NULL | Recruiting | N/A | 65 Years | All | 100 | Phase 2 | United States |
| 94 | JPRN-JapicCTI-090748 | 01/10/2008 | 20/04/2009 | Phase II Study of 0.15% UF-021 in patients with retinitis pigmentosa | Phase II Study of 0.15% UF-021 in patients with retinitis pigmentosa | retinitis pigmentosa | Intervention name : UF-021 (isopropyl unoprostone) Dosage And administration of the intervention : Eye drop | R-Tech Ueno, Ltd | NULL | 20 | 65 | BOTH | 105 | Phase 2 | NULL | |
| 95 | NCT01256697 (ClinicalTrials.gov) | August 2008 | 25/2/2010 | The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions im Patients With Retinitis Pigmentosa | The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella | Retinitis Pigmentosa | Dietary Supplement: Alga Dunaliella Bardawil;Dietary Supplement: Alga Dunaliella Bardawill;Other: Sugar pill | Sheba Medical Center | NULL | Completed | 18 Years | N/A | Female | 34 | N/A | Israel |
| 96 | NCT00661479 (ClinicalTrials.gov) | July 2008 | 29/10/2007 | An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: 400 µg Brimonidine Tartrate Implant;Drug: 200 µg Brimonidine Tartrate Implant;Drug: 100 µg Brimonidine Tartrate Implant;Other: Sham (no implant) | Allergan | NULL | Completed | 18 Years | N/A | All | 21 | Phase 1/Phase 2 | United States;France;Germany;Portugal | |
| 97 | NCT00458575 (ClinicalTrials.gov) | April 2007 | 10/4/2007 | A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis Pigmentosa | A Phase I Open Label Non-comparative Study Evaluating the Safety of a Single, Unilateral, Subretinal Administration of CNTO 2476 in Advanced Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: CNTO 2476 | Centocor, Inc. | NULL | Terminated | 18 Years | N/A | Both | 7 | Phase 1 | United States |
| 98 | NCT00433277 (ClinicalTrials.gov) | February 2007 | 6/2/2007 | Autoimmunity in Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: twice-daily dosage with 0.5% cyclosporine-A eyedrops | Semmelweis University | NULL | Active, not recruiting | N/A | N/A | Both | N/A | Hungary | ||
| 99 | NCT00447980 (ClinicalTrials.gov) | January 2007 | 9/3/2007 | A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa | A Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Field Sensitivity as the Primary Outcome | Retinitis Pigmentosa | Drug: NT-501 | Neurotech Pharmaceuticals | NULL | Completed | 18 Years | 64 Years | Both | 68 | Phase 2 | United States |
| 100 | NCT00447993 (ClinicalTrials.gov) | January 2007 | 9/3/2007 | A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa | A Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Acuity as the Primary Outcome | Retinitis Pigmentosa | Drug: NT-501 | Neurotech Pharmaceuticals | NULL | Completed | 18 Years | 68 Years | Both | 65 | Phase 2 | United States |
| 101 | NCT00065455 (ClinicalTrials.gov) | July 17, 2003 | 23/7/2003 | Investigating the Effect of Vitamin A Supplementation on Retinitis Pigmentosa | Pilot Study on the Effect of Vitamin A Supplementation on Cone Function in Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Vitamin A | National Eye Institute (NEI) | NULL | Completed | 18 Years | N/A | All | 11 | Phase 1 | United States |
| 102 | NCT00346333 (ClinicalTrials.gov) | July 2003 | 27/6/2006 | Clinical Trial of Lutein for Patients With Retinitis Pigmentosa Receiving Vitamin A | Randomized Clinical Trial for Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Lutein;Dietary Supplement: Cornstarch control | National Eye Institute (NEI) | NULL | Completed | 18 Years | 60 Years | All | 240 | Phase 3 | United States |
| 103 | NCT00063765 (ClinicalTrials.gov) | June 2003 | 3/7/2003 | Evaluation of Safety of Ciliary Neurotrophic Factor Implants in the Eye | A Phase I Study of NT-501-10 and NT-501-6A.02, Implants of Encapsulated Human NTC-210 Cells Releasing Ciliary Neurotrophic Factor (CNTF), in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Ciliary Neurotrophic Factor Implant NT-501 | National Eye Institute (NEI) | NULL | Completed | N/A | N/A | Both | 10 | Phase 1 | United States |
| 104 | JPRN-UMIN000007895 | 2001/07/01 | 01/06/2012 | Effects of nilvadipine on the progression of visual field defects in patients with retinitis pigmentosa | Effects of nilvadipine on the progression of visual field defects in patients with retinitis pigmentosa - Effects of nilvadipine on the progression of retinitis pigmentosa | retinitis pigmentosa | Treated group: oral administration of nilvadipine 4mg per day Control group: herenien, tocoferol or no medication as patients' requests | Department of Ophthalmology, Hirosaki University Graduate School of Medicine | NULL | Complete: follow-up complete | 20years-old | 80years-old | Male and Female | 40 | Phase 2 | Japan |
| 105 | NCT00029289 (ClinicalTrials.gov) | April 2001 | 9/1/2002 | Effects of Lutein in Retinitis Pigmentosa | Effects of Lutein in Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Lutein (10 or 30 mg/day) capsules | National Center for Complementary and Integrative Health (NCCIH) | NULL | Completed | Phase 1/Phase 2 | United States | ||||
| 106 | NCT00000116 (ClinicalTrials.gov) | May 1996 | 23/9/1999 | Randomized Trial for Retinitis Pigmentosa | Randomized Trial for Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Vitamin A;Drug: Nutritional Supplement | National Eye Institute (NEI) | NULL | Completed | 18 Years | 56 Years | Both | Phase 3 | United States | |
| 107 | NCT00004827 (ClinicalTrials.gov) | March 1996 | 24/2/2000 | Study of Docosahexaenoic Acid (DHA) Supplementation in Patients With X-Linked Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: docosahexaenoic acid | Retina Foundation of the Southwest | NULL | Completed | N/A | N/A | Male | 46 | N/A | NULL | |
| 108 | NCT00000114 (ClinicalTrials.gov) | May 1984 | 23/9/1999 | Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Vitamin E;Drug: Vitamin A | National Eye Institute (NEI) | NULL | Completed | 18 Years | 49 Years | Both | Phase 3 | NULL | ||
| 109 | EUCTR2021-002729-74-DK (EUCTR) | 26/11/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 81 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Denmark;Netherlands;Norway;Germany;United Kingdom | |||
| 110 | JPRN-JapicCTI-132079 | Phase 3 Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration Period | Phase 3 Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration Period | Retinitis Pigmentosa | Intervention name : UF-021(isopropyl unoprostone) Dosage And administration of the intervention : Eye drop Control intervention name : null | R-Tech Ueno, Ltd | NULL | 20 | 70 | BOTH | Phase 3 | NULL | ||||
| 111 | EUCTR2021-002728-19-NO (EUCTR) | 19/10/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 2;Phase 3 | France;United States;European Union;Canada;Brazil;Denmark;Netherlands;Germany;Norway;United Kingdom | |||
| 112 | EUCTR2020-002255-37-FR (EUCTR) | 05/11/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | NA | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | |||
| 113 | EUCTR2021-002728-19-NL (EUCTR) | 15/01/2022 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Norway;Germany;Netherlands;United Kingdom | |||
| 114 | EUCTR2021-002729-74-NL (EUCTR) | 15/01/2022 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 81 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Norway;Germany;Netherlands;United Kingdom | |||
| 115 | EUCTR2020-002873-88-BE (EUCTR) | 29/10/2021 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR | MeiraGTx UK II Limited | NULL | NA | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | |||
| 116 | EUCTR2020-002255-37-Outside-EU/EEA (EUCTR) | 21/12/2022 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | NA | Female: yes Male: yes | Phase 3 | Canada;Israel;United States;Switzerland;United Kingdom | ||||
| 117 | EUCTR2020-002873-88-Outside-EU/EEA (EUCTR) | 21/12/2022 | Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR | MeiraGTx UK II Limited | NULL | NA | Female: yes Male: yes | Phase 3 | Canada;Israel;United States;Switzerland;United Kingdom | ||||
| 118 | EUCTR2020-002255-37-BE (EUCTR) | 29/10/2021 | Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X. | Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR | X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: AAV5-HRKP.RPGR | MeiraGTx UK II Limited | NULL | NA | Female: yes Male: yes | 66 | Phase 3 | United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands | |||
| 119 | EUCTR2021-002728-19-FR (EUCTR) | 23/09/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;France;European Union;Canada;Brazil;Netherlands;Norway;Germany;United Kingdom | |||
| 120 | EUCTR2021-002729-74-NO (EUCTR) | 14/10/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 81 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Germany;Norway;United Kingdom | |||
| 121 | EUCTR2021-002728-19-DK (EUCTR) | 26/11/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | France;United States;European Union;Canada;Brazil;Denmark;Netherlands;Norway;Germany;United Kingdom |