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
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
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
Participation Requirements
Sex: All
Minimum Age: 8
Maximum Age: 45
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
China
Peking Union Medical College Hospital
RECRUITING
Beijing
Contact Information
Primary
Xinyan Li
Xinyan.li@fronteratherapeutics.com
+86-021-58206061
Backup
Minghui Xue
minghui.xue@fronteratherapeutics.com
+86-021-58206061
Time Frame
Start Date: 2022-11-30
Estimated Completion Date: 2029-11-30
Participants
Target number of participants: 9
Treatments
Experimental: FT-001 Dose 1
Intraocular administration of a single low dose of range FT-001
Experimental: FT-001 Dose 2
Intraocular administration of a single Mid dose of range FT-001
Experimental: FT-001 Dose 3
Intraocular administration of a single High dose of range FT-001
Related Therapeutic Areas
Sponsors
Leads: Frontera Therapeutics