OPA1 Gene Mutation

Clinical trial pipeline · Data from ClinicalTrials.gov

See which OPA1 Gene Mutation trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

RecruitingPhase 1/2

NCT06970106

Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)

This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approxi…

Australia, New Zealand18 participantsStarted 2025-09-30

Treatment: PYC-001

RecruitingNot Applicable

NCT01793168

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…

United States, Australia20,000 participantsStarted 2010-07

All recent trials (4 shown)

Active — Not RecruitingPhase 1

NCT06461286

SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (h…

Australia18 participantsStarted 2024-10-31

Treatment: PYC-001

TerminatedNot Applicable

NCT06140329

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

The purpose of this study is to characterize the disease progression of confirmed OPA1 mutation-associated autosomal dominant optic atrophy (ADOA) by evaluating…

United States, Australia +2 more1 participantsStarted 2024-02-28