Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Recruiting
Phase 1
Phase 2
Interventional Study
Biallelic RPE65 Mutation-associated Retinal Dystrophy
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
- 8 – 45
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 9 (estimated)
- Sponsor
- Frontera Therapeutics · Industry
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
1. Subjects who are able to understand and sign the ICF
2. Female or male aged 8-45 years old when signing the ICF
3. Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Exclusion Criteria:
1. Other interfering eye diseases
2. Presence of any system…
Contacts