2 recruiting, 1 active not recruiting, 1 terminated. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Autosomal Dominant Optic Atrophy: new trials by email Literature Review of Autosomal Dominant Optic Atrophy trials Research Report on Autosomal Dominant Optic Atrophy
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 |
|---|---|---|---|---|---|---|
| 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 | |
| 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 |
Optic Atrophy, Autosomal Dominant (4) · Optic Atrophies, Hereditary (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) · Ataxia Neuropathy Spectrum (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Cranial Nerve Diseases (4) · Eye Diseases (4) · Eye Diseases, Hereditary (4) · Genetic Diseases, Inborn (4) · Heredodegenerative Disorders, Nervous System (4) · Metabolic Diseases (4) · Nervous System Diseases (4) · Neurodegenerative Diseases (4) · Nutritional and Metabolic Diseases (4) · Optic Atrophy (4) · Optic Nerve Diseases (4) · Mitochondrial Diseases (3) · Abnormalities, Multiple (1) · Adrenal Gland Diseases (1)
PYC Therapeutics (3) · Sanford Health (1)
Australia (3) · United States (2) · Austria (1) · France (1) · Germany (1) · Netherlands (1) · New Zealand (1)
Aishwarya Kundu (1) · Sreenivasu Mudumba, PhD (1)