Clinical trial
A Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Name
FT001-C101
Description
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 arms
Trial start
2022-11-30
Estimated PCD
2025-11-30
Trial end
2029-11-30
Status
Recruiting
Phase
Early phase I
Treatment
FT-001 Low Dose
Comparison of different dosages of FT-001
Arms:
FT-001 Dose 1
FT-001 Mid Dose
Comparison of different dosages of FT-001
Arms:
FT-001 Dose 2
FT-001 High Dose
Comparison of different dosages of FT-001
Arms:
FT-001 Dose 3
Size
9
Primary endpoint
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
52 weeks
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 systemic or ocular disease that can cause or likely to cause vision loss
3. There is evidence of obviously uncontrolled concomitant diseases
4. Known to have active or suspected autoimmune diseases
5. With active systemic infection under treatment
6. Pregnant or lactating women
7. Other conditions unsuitable for the study as determined by the investigator
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 9, 'type': 'ESTIMATED'}}
Updated at
2023-05-15
1 organization
1 product
1 indication
Product
FT-001Organization
Frontera Therapeutics