Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
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ClinicalTrials.gov Identifier: NCT03316560 |
Recruitment Status :
Recruiting
First Posted : October 20, 2017
Last Update Posted : July 15, 2021
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Tracking Information | |||||||||
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First Submitted Date ICMJE | October 10, 2017 | ||||||||
First Posted Date ICMJE | October 20, 2017 | ||||||||
Last Update Posted Date | July 15, 2021 | ||||||||
Actual Study Start Date ICMJE | April 16, 2018 | ||||||||
Estimated Primary Completion Date | August 2022 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations | ||||||||
Official Title ICMJE | A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene | ||||||||
Brief Summary | This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations. | ||||||||
Detailed Description | This study includes a non-randomized, open-label, Phase 1/2 dose escalation portion, and a Phase 2 randomized, controlled, masked dose expansion portion. Approximately 30 participants will be enrolled into the dose escalation portion of the study. Each participant will receive the study agent by subretinal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). Within groups 1 through 3 and 5 and 6, enrollment of participants will be staggered by at least 2 weeks to allow adequate time for review of safety information by the investigators and sponsor. Within Group 4, enrollment of the first 3 pediatric participants will be staggered by at least 2 weeks to allow adequate time for review of safety information by the investigators and sponsor. Study agent administration will occur on Day 0. There are a total of 15 visits over approximately 36 months, and long-term follow-up evaluations annually at years 4 and 5. After the phase 1/2 portion of the study is completed, approximately 12 participants, who were not part of the Phase 1/2 portion of the study, will be enrolled into the dose expansion portion of the study. These participants will be randomized in a 1:1 ratio to 1 of 2 treatment groups (i.e., Group 1 [low dose] and Group 2 [high dose]). Each participant will receive the assigned dose of AGTC-501 in one eye on a single occasion. |
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Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Randomized Intervention Model: Single Group Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Masking Description: Neither the investigator nor the subject will know the dose assignment. Both the subject and the investigator will know which eye received treatment. Primary Purpose: Treatment
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Condition ICMJE | X-Linked Retinitis Pigmentosa | ||||||||
Intervention ICMJE | Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
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Study Arms ICMJE |
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Publications * | Not Provided | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status ICMJE | Recruiting | ||||||||
Estimated Enrollment ICMJE |
42 | ||||||||
Original Estimated Enrollment ICMJE |
15 | ||||||||
Estimated Study Completion Date ICMJE | August 2026 | ||||||||
Estimated Primary Completion Date | August 2022 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Phase 1/2 Dose Escalation Inclusion Criteria:
Phase 1/2 Dose Escalation Exclusion Criteria: • Have other known retinal disease mutations or previously received an AAV gene therapy product, as well as being unable or unwilling to meet the requirements of the study Phase 2 Dose Expansion Inclusion Criteria:
Phase 2 Dose Expansion Exclusion Criteria • Have other known retinal disease mutations or previously received an AAV gene therapy product, as well as being unable or unwilling to meet the requirements of the study |
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Years to 50 Years (Child, Adult) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | United States | ||||||||
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Administrative Information | |||||||||
NCT Number ICMJE | NCT03316560 | ||||||||
Other Study ID Numbers ICMJE | AGTC-RPGR-001 | ||||||||
Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Applied Genetic Technologies Corp | ||||||||
Original Responsible Party | Same as current | ||||||||
Current Study Sponsor ICMJE | Applied Genetic Technologies Corp | ||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||
Collaborators ICMJE | Not Provided | ||||||||
Investigators ICMJE |
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PRS Account | Applied Genetic Technologies Corp | ||||||||
Verification Date | March 2021 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |