SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
NCT06461286 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 18
Last updated 2026-01-21
Summary
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
Conditions
- OPA1 Gene Mutation
- Autosomal Dominant Optic Atrophy
- Hereditary Optic Atrophies
- Kjer Optic Atrophy
Interventions
- DRUG
-
PYC-001
Single Group Assignment
Sponsors & Collaborators
-
PYC Therapeutics
lead INDUSTRY
Principal Investigators
-
Sreenivasu Mudumba, PhD · PYC Therapeutics
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-10-31
- Primary Completion
- 2026-08-31
- Completion
- 2026-08-31
Countries
- Australia
Study Locations
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