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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05858983
Recruitment Status : Recruiting
First Posted : May 15, 2023
Last Update Posted : May 15, 2023
Sponsor:
Information provided by (Responsible Party):
Frontera Therapeutics

Tracking Information
First Submitted Date  ICMJE April 29, 2023
First Posted Date  ICMJE May 15, 2023
Last Update Posted Date May 15, 2023
Actual Study Start Date  ICMJE November 30, 2022
Estimated Primary Completion Date November 30, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 11, 2023)		 Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) [ Time Frame: 52 weeks ]
Incidence of ocular and non-ocular AEs and SAEs
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: May 11, 2023)
  • Changes in visual function from baseline [ Time Frame: 52 weeks ]
    Changes in visual function from baseline as assessed by FST
  • Changes in visual function from baseline [ Time Frame: 52 weeks ]
    Changes in visual function from baseline as assessed by Mobility courses
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Biallelic RPE65 Mutation-associated Retinal Dystrophy
Intervention  ICMJE
  • Genetic: FT-001 Low Dose
    Comparison of different dosages of FT-001
  • Genetic: FT-001 Mid Dose
    Comparison of different dosages of FT-001
  • Genetic: FT-001 High Dose
    Comparison of different dosages of FT-001
Study Arms  ICMJE
  • Experimental: FT-001 Dose 1
    Intraocular administration of a single low dose of range FT-001
    Intervention: Genetic: FT-001 Low Dose
  • Experimental: FT-001 Dose 2
    Intraocular administration of a single Mid dose of range FT-001
    Intervention: Genetic: FT-001 Mid Dose
  • Experimental: FT-001 Dose 3
    Intraocular administration of a single High dose of range FT-001
    Intervention: Genetic: FT-001 High Dose
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 11, 2023)		 9
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 30, 2029
Estimated Primary Completion Date November 30, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 45 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Xinyan Li +86-021-58206061 Xinyan.li@fronteratherapeutics.com
Contact: Minghui Xue +86-021-58206061 minghui.xue@fronteratherapeutics.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05858983
Other Study ID Numbers  ICMJE FT001-C101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Frontera Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Frontera Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Ruifang Sui Peking Union Medical College Hospital
PRS Account Frontera Therapeutics
Verification Date October 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP