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

• Sponsor: Frontera Therapeutics (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05858983
• Collaborator: (none)
• Enrollment: 9
• Location: 1
• Arms: 3
• Anticipated Duration (Months): 84
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Biallelic RPE65 Mutation-associated Retinal Dystrophy	Genetic: FT-001 Low Dose; Genetic: FT-001 Mid Dose; Genetic: FT-001 High Dose	Phase 1/Phase 2

Detailed 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.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 9 participants
• Allocation: Non-Randomized
• Intervention Model: Sequential Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Nov 30, 2022
• Anticipated Primary Completion Date: Nov 30, 2025
• Anticipated Study Completion Date: Nov 30, 2029

Arms and Interventions

Arm	Intervention/Treatment
Experimental: FT-001 Dose 1 Intraocular administration of a single low dose of range FT-001	Genetic: FT-001 Low Dose; Comparison of different dosages of FT-001
Experimental: FT-001 Dose 2 Intraocular administration of a single Mid dose of range FT-001	Genetic: FT-001 Mid Dose; Comparison of different dosages of FT-001
Experimental: FT-001 Dose 3 Intraocular administration of a single High dose of range FT-001	Genetic: FT-001 High Dose; Comparison of different dosages of FT-001

Outcome Measures

Primary Outcome Measures

1. Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) [52 weeks]

   Incidence of ocular and non-ocular AEs and SAEs

Secondary Outcome Measures

1. Changes in visual function from baseline [52 weeks]

   Changes in visual function from baseline as assessed by FST

2. Changes in visual function from baseline [52 weeks]

   Changes in visual function from baseline as assessed by Mobility courses

Eligibility Criteria

Criteria

• Ages Eligible for Study: 8 Years to 45 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Peking Union Medical College Hospital	Beijing	Beijing	China	100142

Sponsors and Collaborators

• Frontera Therapeutics

Investigators

• Principal Investigator: Ruifang Sui, Peking Union Medical College Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Frontera Therapeutics
• ClinicalTrials.gov Identifier: NCT05858983
• Other Study ID Numbers: FT001-C101
• First Posted: May 15, 2023
• Last Update Posted: May 15, 2023
• Last Verified: Oct 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Frontera Therapeutics: FT-001; Biallelic RPE65 Mutation-associated Retinal Dystrophy
• Additional relevant MeSH terms: Retinal Dystrophies

Study Results

No Results Posted as of May 15, 2023