4 recruiting, 1 active not recruiting, 1 completed, 1 terminated. 2 Not applicable, 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 2 | Completed 1, Recruiting 1 |
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Optic Atrophy, Autosomal Dominant: new trials by email Literature Review of Optic Atrophy, Autosomal Dominant trials Research Report on Optic Atrophy, Autosomal Dominant
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Longitudinal Natural History Study of OPA1-Associated Autosomal-Dominant Optic Atrophy OPA1 Gene Mutation, Optic Atrophy, Autosomal Dominant | Recruiting | Ludwig-Maximilians - University of Munich | 50 | 2026-07-16 | 2026-07-28 | |
| Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle) PYC-001 - OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy | Phase 1 / Phase 2 | Recruiting | PYC Therapeutics | 24 | 2025-09-30 | 2026-06-29 |
| SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew) PYC-001 - OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy | Phase 1 | Active not recruiting | PYC Therapeutics | 18 | 2024-10-31 | 2026-09-17 |
| Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant | Terminated | PYC Therapeutics | 1 | 2024-02-28 | 2025-03-14 | |
| Stem Cell Ophthalmology Treatment Study II Arm 1 - Retinal Disease, Age-Related Macular Degeneration | Not applicable | Recruiting | MD Stem Cells | 500 | 2016-01-01 | 2026-06-29 |
| Advanced Characterization of Autosomal Dominant Optic Atrophy Optic Atrophy, Autosomal Dominant | Unknown | Glostrup University Hospital, Copenhagen | 50 | 2011-12-01 | 2012-01-31 | |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2026-10-28 | |
| Transcorneal Electrical Stimulation Therapy for Retinal Disease Transcorneal Electrical Stimulation, DTL-electrode attached without energy - Retinitis Pigmentosa, Macula Off | Not applicable | Completed | Okuvision GmbH | 80 | 2008-01-01 | 2012-05-04 |
Optic Atrophies, Hereditary (3) · Retinitis Pigmentosa (3) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Activated PI3K-delta Syndrome (1) · Addison Disease (1) · Alagille Syndrome (1) · Alstrom Syndrome (1) · Amnesia, Transient Global (1) · Aniridia (1) · Aniridia and Absent Patella (1) · Aniridia, Partial, with Unilateral Renal Agenesis and Psychomotor Retardation (1) · Ataxia (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Cranial Nerve Diseases (8) · Eye Diseases, Hereditary (8) · Genetic Diseases, Inborn (8) · Heredodegenerative Disorders, Nervous System (8) · Metabolic Diseases (8) · Nervous System Diseases (8) · Neurodegenerative Diseases (8) · Nutritional and Metabolic Diseases (8) · Eye Diseases (7) · Mitochondrial Diseases (7) · Optic Atrophy (7) · Optic Nerve Diseases (7) · Optic Atrophies, Hereditary (5) · Cardiovascular Diseases (3)
PYC Therapeutics (3) · Glostrup University Hospital, Copenhagen (1) · Ludwig-Maximilians - University of Munich (1) · MD Stem Cells (1) · Okuvision GmbH (1) · Sanford Health (1)
Australia (3) · United States (3) · Austria (2) · Germany (2) · Denmark (1) · France (1) · Netherlands (1) · New Zealand (1) · United Arab Emirates (1)
Aishwarya Kundu (1) · Florian Gekeler, MD, Prof (1) · Jeffrey Weiss, MD (1) · Maximilian-Joachim Gerhardt, Dr. med. (1) · Michael Larsen, MD, Prof. DMSc (1) · Sreenivasu Mudumba, PhD (1) · Steven Levy, MD (1)
PMID 21467183, PMID 23049087, PMID 25186535, PMID 26199618, PMID 26604914, PMID 27857759, PMID 27904503, PMID 29270420, PMID 30050918, PMID 31119146, PMID 31620478, PMID 32039263, PMID 32231088, PMID 33546345