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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

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : November 30, 2022
Estimated Primary Completion Date : November 30, 2025
Estimated Study Completion Date : November 30, 2029
Arms and Interventions
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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
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Changes in visual function from baseline [ Time Frame: 52 weeks ]
    Changes in visual function from baseline as assessed by FST

  2. Changes in visual function from baseline [ Time Frame: 52 weeks ]
    Changes in visual function from baseline as assessed by Mobility courses


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05858983


Contacts
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Contact: Xinyan Li +86-021-58206061 Xinyan.li@fronteratherapeutics.com
Contact: Minghui Xue +86-021-58206061 minghui.xue@fronteratherapeutics.com

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100142
Contact: Ruifang Sui, Professor         
Sponsors and Collaborators
Frontera Therapeutics
Investigators
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Principal Investigator: Ruifang Sui Peking Union Medical College Hospital
More Information
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Responsible Party: Frontera Therapeutics
ClinicalTrials.gov Identifier: NCT05858983    
Other Study ID Numbers: FT001-C101
First Posted: May 15, 2023    Key Record Dates
Last Update Posted: May 15, 2023
Last Verified: October 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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Retinal Dystrophies
Retinal Degeneration
Retinal Diseases
Eye Diseases