Autosomal Dominant Optic Atrophy

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Autosomal Dominant Optic Atrophy trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 3 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

RecruitingNot Applicable

NCT03011541

Stem Cell Ophthalmology Treatment Study II

This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.

United States, Austria, United Arab Emirates500 participantsStarted 2016-01

Treatment: Arm 1

All recent trials (11 shown)

Active — Not RecruitingPhase 2/3

NCT06007391

Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1

Dominant Optic Atrophy (hereafter known as DOA) is a neurodegenerative pathology of the optic nerve inducing progressive loss of central visual field and visual…

France25 participantsStarted 2024-01-23

Treatment: Nicotinamide

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

Autosomal Dominant Optic Atrophy

Trial phase breakdown

Trial status

Actively recruiting — 3 trials

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

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

Stem Cell Ophthalmology Treatment Study II

All recent trials (11 shown)

Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1

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

Trial phase breakdown

Trial status

Actively recruiting — 3 trials

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

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

Stem Cell Ophthalmology Treatment Study II

All recent trials (11 shown)

Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1

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

Misfolded Proteins in the Skin of People With Parkinson's Disease and Other Parkinsonism

A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathi…

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

Biomarkers in Parkinsonian Syndromes

Transcorneal Electrical Stimulation Therapy for Retinal Disease

Advanced Characterization of Autosomal Dominant Optic Atrophy