Back to resultsGene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Primary Purpose
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
FT-001 Low Dose
FT-001 Mid Dose
FT-001 High Dose
Sponsored by
About this trial
This is an interventional treatment trial for Biallelic RPE65 Mutation-associated Retinal Dystrophy focused on measuring FT-001, Biallelic RPE65 Mutation-associated Retinal Dystrophy
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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 With active systemic infection under treatment Pregnant or lactating women Other conditions unsuitable for the study as determined by the investigator
Sites / Locations
- Peking Union Medical College HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
FT-001 Dose 1
FT-001 Dose 2
FT-001 Dose 3
Arm Description
Intraocular administration of a single low dose of range FT-001
Intraocular administration of a single Mid dose of range FT-001
Intraocular administration of a single High dose of range FT-001
Outcomes
Primary Outcome Measures
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Incidence of ocular and non-ocular AEs and SAEs
Secondary Outcome Measures
Changes in visual function from baseline
Changes in visual function from baseline as assessed by FST
Changes in visual function from baseline
Changes in visual function from baseline as assessed by Mobility courses
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05858983
Brief Title
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
November 30, 2022 (Actual)
Primary Completion Date
November 30, 2025 (Anticipated)
Study Completion Date
November 30, 2029 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Frontera Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Keywords
FT-001, Biallelic RPE65 Mutation-associated Retinal Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
9 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
FT-001 Dose 1
Arm Type
Experimental
Arm Description
Intraocular administration of a single low dose of range FT-001
Arm Title
FT-001 Dose 2
Arm Type
Experimental
Arm Description
Intraocular administration of a single Mid dose of range FT-001
Arm Title
FT-001 Dose 3
Arm Type
Experimental
Arm Description
Intraocular administration of a single High dose of range FT-001
Intervention Type
Genetic
Intervention Name(s)
FT-001 Low Dose
Intervention Description
Comparison of different dosages of FT-001
Intervention Type
Genetic
Intervention Name(s)
FT-001 Mid Dose
Intervention Description
Comparison of different dosages of FT-001
Intervention Type
Genetic
Intervention Name(s)
FT-001 High Dose
Intervention Description
Comparison of different dosages of FT-001
Primary Outcome Measure Information:
Title
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Description
Incidence of ocular and non-ocular AEs and SAEs
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Changes in visual function from baseline
Description
Changes in visual function from baseline as assessed by FST
Time Frame
52 weeks
Title
Changes in visual function from baseline
Description
Changes in visual function from baseline as assessed by Mobility courses
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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
With active systemic infection under treatment
Pregnant or lactating women
Other conditions unsuitable for the study as determined by the investigator
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xinyan Li
Phone
+86-021-58206061
Email
Xinyan.li@fronteratherapeutics.com
First Name & Middle Initial & Last Name or Official Title & Degree
Minghui Xue
Phone
+86-021-58206061
Email
minghui.xue@fronteratherapeutics.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ruifang Sui
Organizational Affiliation
Peking Union Medical College Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100142
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ruifang Sui, Professor
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
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