2 recruiting, 1 active not recruiting. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow OPA1 Gene Mutation: new trials by email Literature Review of OPA1 Gene Mutation trials Research Report on OPA1 Gene Mutation
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 |
Optic Atrophy, Autosomal Dominant (3) · Optic Atrophies, Hereditary (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Cranial Nerve Diseases (3) · Eye Diseases (3) · Eye Diseases, Hereditary (3) · Genetic Diseases, Inborn (3) · Heredodegenerative Disorders, Nervous System (3) · Metabolic Diseases (3) · Mitochondrial Diseases (3) · Nervous System Diseases (3) · Neurodegenerative Diseases (3) · Nutritional and Metabolic Diseases (3) · Optic Atrophy (3) · Optic Nerve Diseases (3) · Optic Atrophies, Hereditary (1)
PYC Therapeutics (2) · Ludwig-Maximilians - University of Munich (1)
Australia (2) · Germany (1) · New Zealand (1)
Aishwarya Kundu (1) · Maximilian-Joachim Gerhardt, Dr. med. (1)