Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age8-45
SponsorFrontera Therapeutics
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.
Eligibility criteria
Qualifiers
Subjects who are able to understand and sign the ICF
Female or male aged 8-45 years old when signing the ICF
Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Disqualifiers
Other interfering eye diseases
Presence of any systemic or ocular disease that can cause or likely to cause vision loss
There is evidence of obviously uncontrolled concomitant diseases
Known to have active or suspected autoimmune diseases
Trial design
Treatments tested in this trial
- FT-001 Low Dose
- FT-001 Mid Dose
- FT-001 High Dose
Treatment groups
9 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov