Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Biallelic RPE65 Mutation-associated Retinal Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05858983
• Other study ID #: FT001-C101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: November 30, 2022
• Est. completion date: November 30, 2029

Study information

• Verified date: October 2022
• Source: Frontera Therapeutics
• Contact: Xinyan Li
• Phone: +86-021-58206061
• Email: Xinyan.li[@]fronteratherapeutics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 9
• Est. completion date: November 30, 2029
• Est. primary completion date: November 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Biallelic RPE65 Mutation-associated Retinal Dystrophy
• Retinal Dystrophies

Intervention

Genetic:
• FT-001 Low Dose
Comparison of different dosages of FT-001
• FT-001 Mid Dose
Comparison of different dosages of FT-001
• FT-001 High Dose
Comparison of different dosages of FT-001

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Frontera Therapeutics

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)	Incidence of ocular and non-ocular AEs and SAEs	52 weeks
Secondary	Changes in visual function from baseline	Changes in visual function from baseline as assessed by FST	52 weeks
Secondary	Changes in visual function from baseline	Changes in visual function from baseline as assessed by Mobility courses	52 weeks