90. 網膜色素変性症 Retinitis pigmentosa Clinical trials / Disease details
臨床試験数 : 130 / 薬物数 : 180 - (DrugBank : 41) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 109
Showing 1 to 10 of 130 diseases
| 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 | NCT05282953 (ClinicalTrials.gov) | August 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 Patient s With Retinitis Pigmentosa (ABACUS) A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-30 ... | A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patient s With Retinitis Pigmentosa (ABACUS) A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-30 ... | Retinitis Pigmentosa | Drug: KIO-301 | Kiora Pharmaceuticals, Inc. | NULL | Not yet recruiting | 18 Years | 80 Years | All | 6 | Phase 1 | NULL |
| 2 | 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. Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa(progressive r ... | 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 Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis PigmentosaAssociat ... | 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] X-Linked Retinitis Pigmentosacaused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cla ... | 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 Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | 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 Switzerland;United Kingdom;France;United States;Spain;Ireland;Israel;Italy;Canada;Belgium;Denmark;Ne ... | ||
| 3 | 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. Gene Therapy Trial for Patients with Retinitis Pigmentosa(progressive reduction in vision) due to a ... | 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 Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigme ... | 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] X-Linked Retinitis Pigmentosacaused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cla ... | 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 Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | 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 United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Ne ... | ||
| 4 | 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. Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa(progressive r ... | 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 Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis PigmentosaAssociat ... | 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] X-Linked Retinitis Pigmentosacaused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cla ... | 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 Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | 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 United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Ge ... | ||
| 5 | 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. Gene Therapy Trial for Patients with Retinitis Pigmentosa(progressive reduction in vision) due to a ... | 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 Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigme ... | 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] X-Linked Retinitis Pigmentosacaused by mutations in the RPGR gene MedDRA version: 20.0;Level: PT;Cla ... | 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 Product Name: AAV5-hRKp.RPGR INN or Proposed INN: botaretigene sparoparvovec Other descriptive name: A ... | 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 United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Ge ... | ||
| 6 | NCT05203939 (ClinicalTrials.gov) | January 24, 2022 | 16/11/2021 | The Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa | A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 (Nuclear Receptor Subfamily 2 Group E Member 3) and RHO (Rhodopsin) Mutations A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis PigmentosaAssociated Wit ... | Retinitis Pigmentosa | Drug: OCU400 Low Dose;Drug: OCU400 Mid Dose;Drug: OCU400 High Dose | Ocugen | NULL | Recruiting | 18 Years | N/A | All | 18 | Phase 1/Phase 2 | United States |
| 7 | NCT05147701 (ClinicalTrials.gov) | January 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 Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for the Treatment ... | Eye Diseases;Retinitis Pigmentosa;Glaucoma;Diabetic Retinopathy;Macular Degeneration;Traumatic Optic Neuropathy;Optic Atrophy Eye Diseases;Retinitis Pigmentosa;Glaucoma;Diabetic Retinopathy;Macular Degeneration;Traumatic Optic ... | Biological: AlloRx | The Foundation for Orthopaedics and Regenerative Medicine | NULL | Recruiting | N/A | N/A | All | 20 | Phase 1 | Antigua and Barbuda |
| 8 | 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) Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutatio ... | 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 A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and To ... | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Her ... | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Recruiting | 12 Years | N/A | All | 120 | Phase 2/Phase 3 | United States;United Kingdom |
| 9 | NCT05158296 (ClinicalTrials.gov) | December 8, 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 Advanced Vision Loss (Sirius) Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutatio ... | 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 A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and To ... | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Her ... | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Recruiting | 12 Years | N/A | All | 81 | Phase 2/Phase 3 | United States;United Kingdom |
| 10 | NCT05085964 (ClinicalTrials.gov) | September 13, 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 Safety & Tolerability of QR-421a in Subjects With Retiniti ... | 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) An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Re ... | Retinitis Pigmentosa;Usher Syndrome Type 2 | Drug: RNA antisense oligonucleotide for intravitreal injection | ProQR Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | All | 20 | Phase 2 | United States;Canada;France |