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
This trial does not accept healthy volunteersQualifiers
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
Design model
Sequential
Treatments tested in this trial
FT-001 Low Dose
GeneticComparison of different dosages of FT-001
FT-001 Mid Dose
GeneticComparison of different dosages of FT-001
FT-001 High Dose
GeneticComparison of different dosages of FT-001
Treatment groups
9 Participants
are divided into 3 treatment groupsGroup A: FT-001 Dose 1Experimental treatment 1 intervention
Group B: FT-001 Dose 2Experimental treatment 1 intervention
Group C: FT-001 Dose 3Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Incidence of ocular and non-ocular AEs and SAEs
Time frame
52 weeksSecondary outcomes
1
Changes in visual function from baseline
Changes in visual function from baseline as assessed by FST
Time frame
52 weeks2
Changes in visual function from baseline
Changes in visual function from baseline as assessed by Mobility courses
Time frame
52 weeksSponsors and contacts
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Source ClinicalTrials.gov