Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
Recruiting
Phase 1
Phase 2
Interventional Study
OPA1 Gene Mutation
Autosomal Dominant Optic Atrophy
Hereditary Optic Atrophies
Kjer Optic Atrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 24 (estimated)
- Sponsor
- PYC Therapeutics · Industry
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About This Trial
This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approximately 18 participants from Australia, New Zealand, and other APAC countries are expected to be enrolled, depending on safety review committee (SRC) throughout the course of the study.
Participants may be assigned to any of the following:
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Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Must give written informed consent before any study-related activity is carried out
2. Adult males and females, aged 18 years and above at screening;
3. Body mass index ≥18.0 and ≤35.0 kg/m2
4. Have a recent (within five years) genetic diagnosis of OPA1 mutation-associated (h…
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