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A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

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: NCT04850118
Recruitment Status : Not yet recruiting
First Posted : April 20, 2021
Last Update Posted : May 21, 2021
Sponsor:
Information provided by (Responsible Party):
Applied Genetic Technologies Corp

Study Description
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Brief Summary:
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501) to an untreated control group in male patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Biological: rAAV2tYF-GRK1-hRPGRco Phase 2 Phase 3

Detailed Description:

This Phase 2/3 study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1(low dose) or Dose 2(high dose) will be administered in subjects in 2 treatment groups (N=~42), while subjects in the untreated control group (N=~21) will be followed and evaluated up to Month 12, after which they will be eligible to receive treatment with AGTC-501 Dose 2.

Approximately 63 eligible male subjects between 8 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups (low dose, high dose, untreated control).

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Subjects are randomized in a 1:1:1 ratio to 1 of 3 groups (low dose, high dose, untreated). After 12 months, subjects in the untreated group are eligible to receive the high dose of AGTC-501.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: For groups 1 and 2 (AGTC-501 dosing groups), both the subjects and the Investigators are masked to dose assignment, and only the unmasked staff who prepare the dose are unmasked to the subject's dose assignment. Due to the need to perform the subretinal injection to administer the study drug, both subjects and Investigators will know whether the subject was assigned to a treatment group or to the control group. To minimize bias of the treated and control study eye evaluations, starting at Month 3, microperimetry, mobility testing, and BCVA assessments will be conducted by appropriately qualified masked evaluators who do not know whether the subject underwent surgery. Subjects will be instructed not to disclose whether they had surgery to the examiner administering the test.
Primary Purpose: Treatment
Official Title: A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
Estimated Study Start Date : August 2021
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : March 2029

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Group 1: Low Dose
Male subjects 8-50 years of age treated by subretinal injection with the low dose of AGTC-501
 Biological: rAAV2tYF-GRK1-hRPGRco
Adeno-associated virus vector expressing a human RPGR gene
Other Name: AGTC-501
Active Comparator: Group 2: High Dose
Male subjects 8-50 years of age treated by subretinal injection with the high dose of AGTC-501
 Biological: rAAV2tYF-GRK1-hRPGRco
Adeno-associated virus vector expressing a human RPGR gene
Other Name: AGTC-501
Group 3: Control
Male subjects 8-50 years of age in the untreated control group. Subjects randomized to the untreated control group will be followed for a minimum of 12 months. After this time, they will be treated by subretinal injection with the high dose of AGTC-501.
 Biological: rAAV2tYF-GRK1-hRPGRco
Adeno-associated virus vector expressing a human RPGR gene
Other Name: AGTC-501


Outcome Measures
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Primary Outcome Measures :
  1. The difference in the proportion of responding eyes between treated and control eyes in low dose group and high dose group [ Time Frame: Day 0 - Month 12 ]

Secondary Outcome Measures :
  1. - Proportion of responding eyes in treated study eyes in the low dose group and high dose group versus untreated control eyes, as measured by mobility test score change [ Time Frame: Day 0 - Month 12 ]
  2. - Proportion of responding eyes in treated eyes versus control eyes in the low dose and high dose group measured by visual function assessments [ Time Frame: Day 0 - Month 12 ]
  3. - Difference in mean change from baseline in Visual Acuity in treated eyes versus control eyes in the low dose and high dose groups [ Time Frame: Day 0 - Month 12 ]
  4. - Difference in mean change from baseline in the EZ area, as measured by SD-OCT, in treated study eyes in the low dose group and high dose group versus untreated control study eyes [ Time Frame: Day 0 - Month 12 ]
  5. Changes from baseline in quality of life questionnaire responses [ Time Frame: Day 0 - Month 12 ]

Other Outcome Measures:
  1. Number and proportion of treatment-emergent adverse events [ Time Frame: Day 0 - Year 5 ]
  2. Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters [ Time Frame: Day 0 - Year 5 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males between the ages of 8-50 years old with a clinical diagnosis of XLRP with a confirmed RPGR mutation who also meet the other requirements of the study.
  • Have at least one documented pathogenic or likely pathogenic variant in the RPGR gene within exons 1-14 and/or ORF15
  • Have a BCVA no better than 75 letters (20/32) and no worse than 35 letters (20/200) in the study eye based on an Early Treatment of Diabetic Retinopathy Study (ETDRS) chart at each screening visit

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.

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): NCT04850118


Contacts
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Contact: Serva Health 855-467-2364 ProviderSupport@scenictrials.com

Sponsors and Collaborators
Applied Genetic Technologies Corp
Investigators
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Study Director: Matthew Feinsod, MD Applied Genetic Technologies Corporation
More Information
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Responsible Party: Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier: NCT04850118    
Other Study ID Numbers: AGTC-RPGR-002
First Posted: April 20, 2021    Key Record Dates
Last Update Posted: May 21, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Applied Genetic Technologies Corp:
XLRP
retinal degeneration
RPGR
 adeno-associated virus
AAV
gene therapy
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn