VA ( DrugBank: - )
2 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 90 | Retinitis pigmentosa | 24 |
| 302 | Leber hereditary optic neuropathy | 3 |
90. Retinitis pigmentosa
Clinical trials : 147 / Drugs : 176 - (DrugBank : 43) / Drug target genes : 49 - Drug target pathways : 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 | NCT05537220 (ClinicalTrials.gov) | June 2023 | 8/9/2022 | Oral N-acetylcysteine for Retinitis Pigmentosa | NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: N-acetylcysteine;Drug: Placebo | Johns Hopkins University | National Eye Institute (NEI);Duke University;Emory University;Massachusetts Eye and Ear Infirmary;Mayo Clinic;Medical College of Wisconsin;Retina Foundation of the Southwest;Stanford University;University of California, Davis;University of Florida;University of Illinois at Chicago;University of Iowa;University of Miami;University of Michigan;University of Minnesota;University of Oklahoma;University of Southern California;University of Utah;University of Washington;University of Wisconsin, Madison;Vanderbilt University;Vitreo Retinal Associates, PA;University of Houston;Medical University of Graz;McGill University;Universität Tübingen;Centro Medico ABC;Radboud University Medical Center;University of Amsterdam;University Hospital, Basel, Switzerland;University College London Hospitals;Northwestern University | Not yet recruiting | 18 Years | 65 Years | All | 438 | Phase 3 | United States;Austria;Canada;Germany;Mexico;Netherlands;Switzerland;United Kingdom |
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 |
| 11 | NCT04671433 (ClinicalTrials.gov) | March 16, 2021 | 5/11/2020 | Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | X-Linked Retinitis Pigmentosa | Biological: Genetic: AAV5-RPGR | MeiraGTx UK II Ltd | Janssen Research & Development, LLC | Recruiting | 3 Years | N/A | All | 96 | Phase 3 | United States;Denmark;France;Israel;Netherlands;Spain;Switzerland;United Kingdom |
| 12 | NCT04794101 (ClinicalTrials.gov) | March 16, 2021 | 15/12/2020 | Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | Follow-up Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | X-Linked Retinitis Pigmentosa | Biological: Genetic: AAV5-RPGR 4e11;Biological: Genetic: AAV5-RPGR 2e11 | MeiraGTx UK II Ltd | Janssen Research & Development, LLC | Recruiting | 3 Years | N/A | All | 96 | Phase 3 | United States;Denmark;France;Israel;Netherlands;Spain;Switzerland;United Kingdom |
| 13 | 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 | |
| 14 | 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 |
| 15 | 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 |
| 16 | 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 |
| 17 | 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 | |
| 18 | JPRN-JMA-IIA00053 | 24/12/2010 | 22/11/2010 | Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa | Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa | Retinitis Pigmentosa | Intervention type:DRUG. Intervention1:Sodium valproate, Dose form:TABLET, Route of administration:ORAL. | Yasuhiko Hirami | Masayo Takahashi, Yasuo Kurimoto | Completed | >=20 YEARS | No Limit | BOTH | 30 | NOT APPLICABLE | Japan |
| 19 | NCT01233609 (ClinicalTrials.gov) | November 2010 | 1/11/2010 | Trial of Oral Valproic Acid for Retinitis Pigmentosa | A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis Pigmentosa | Retinitis Pigmentosa | Drug: Valproic Acid;Drug: Placebo | Foundation Fighting Blindness Clinical Research Institute | United States Department of Defense | Completed | 18 Years | N/A | All | 90 | Phase 2 | United States |
| 20 | 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 | |||
| 21 | 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 | ||||
| 22 | 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 | ||||
| 23 | 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 | |||
| 24 | 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 |
302. Leber hereditary optic neuropathy
Clinical trials : 23 / Drugs : 15 - (DrugBank : 5) / Drug target genes : 5 - Drug target pathways : 33
| 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 | EUCTR2015-001266-26-IT (EUCTR) | 18/03/2016 | 10/05/2019 | A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 months | Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo maggiore di 6 mesi e fino a 12 mesi da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - REVERSE | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene Product Code: GS010 Other descriptive name: RAAV2/2-ND4 VECTOR | GENSIGHT BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2015-001265-11-IT (EUCTR) | 18/03/2016 | 04/11/2020 | A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less | Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo di 6 mesi o inferiore, da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - RESCUE | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene Product Code: GS010 INN or Proposed INN: PENDING Other descriptive name: RAAV2/2-ND4 VECTOR | GENSIGHT BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;France;Germany;United Kingdom;Italy | ||
| 3 | JPRN-UMIN000017939 | 2013/10/01 | 20/06/2015 | Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy | Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy - Clinical trial of Idebenone in patients with LHON | Leber hereditary optic neuropathy | 1. Clinical trial medicine: Idebenone 900mg/day 2. Objectives: 50 patients with LHON 3. Exclusion criteria: a) A smoker b) A patient with abnormality of hepatic function c) A patient who present seizures, delirium and hallucination d) Pregnancy or Lactation e) A patient who is associated with agranulocytosis f) A patient with chronic renal failure g) A patient with anaphylactic shock against Idebenone 4. Duration of drug administration: 6 months 5. Examinations schedules: Both subjective and objective examinations are performed as following schedules; a) At the base line: Visual acuity (VA), Critical flicker frequency (CFF), Visual field (VF: Humphry 30-2), central retinal thickness (CRT), functional MRI (f-MRI), searching the mitochondrial mutation b) 8 weeks: VA, CFF, VF, CRT, f-MRI c) 16 weeks: VA, CFF, VF, CRT, f-MRI d) 24 weeks: VA, CFF, VF, CRT, f-MRI e) 32 weeks: VA, CFF, VF, CRT, f-MRI f) 40 weeks: VA, CFF, VF, CRT, f-MRI g) 48 weeks: VA, CFF, VF, CRT, f-MRI | Hyogo College of Medicine | Kitasato UniversityJikei University School of MedicineTokyo Medical University | Complete: follow-up complete | 10years-old | 80years-old | Male and Female | 50 | Phase 1,2 | Japan |