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History of Changes for Study: NCT03316560
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR-ORF15 Mutations
Latest version (submitted July 13, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 October 17, 2017 None (earliest Version on record)
2 January 2, 2018 Recruitment Status, Study Status, Contacts/Locations and Oversight
3 December 10, 2018 Contacts/Locations, Study Status, Study Identification, Eligibility and Study Description
4 May 22, 2019 Arms and Interventions, Study Status, Contacts/Locations, Study Description, Eligibility, Outcome Measures, Study Design and Conditions
5 December 9, 2019 Contacts/Locations and Study Status
6 November 30, 2020 Study Status
7 January 27, 2021 Contacts/Locations, Outcome Measures, Arms and Interventions, Study Design, Study Status, Eligibility and Study Description
8 March 10, 2021 Study Status and Contacts/Locations
9 June 15, 2021 Contacts/Locations, Study Description, Eligibility, Outcome Measures, Study Design, Study Status and Study Identification
10 July 13, 2021 Contacts/Locations and Study Status
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Study NCT03316560
Submitted Date:  October 17, 2017 (v1)

Open or close this module Study Identification
Unique Protocol ID: AGTC-RPGR-001
Brief Title: Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR-ORF15 Mutations
Official Title: An Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR-ORF15 Mutations
Secondary IDs:
Open or close this module Study Status
Record Verification: October 2017
Overall Status: Not yet recruiting
Study Start: January 2018
Primary Completion: January 2022 [Anticipated]
Study Completion: January 2024 [Anticipated]
First Submitted: October 10, 2017
First Submitted that
Met QC Criteria:
October 17, 2017
First Posted: October 20, 2017 [Actual]
Last Update Submitted that
Met QC Criteria:
October 17, 2017
Last Update Posted: October 20, 2017 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Applied Genetic Technologies Corp
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing a human RPGR-ORF15 gene (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR-ORF15 mutations. Approximately 15 participants will be enrolled and 3 dose levels will be evaluated in a dose-escalation format.
Detailed Description:

This will be a non-randomized, open-label, Phase 1/2 dose escalation study.

Approximately 15 participants will be enrolled. 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). Participants in the dose escalation phase will be ≥ 18 years of age. After the maximum tolerated dose is identified individuals ≥ 6 years of age will be enrolled.

Safety will be measured by the number and proportion of participants experiencing ocular and non-ocular adverse events or abnormal clinically relevant hematology or clinical chemistry parameters. Efficacy will be measured by evaluation of changes in visual structure, function, and quality of life.

Open or close this module Conditions
Conditions: X-Linked Retinitis Pigmentosa
Keywords: XLRP
retinal degeneration
RPGR
ORF15
adeno-associated virus
gene therapy
AAV
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms: 4
Masking: None (Open Label)
Allocation: Non-Randomized
Enrollment: 15 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Group 1
Subjects at least 18 y/o treated with a lower dose of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR-ORF15 gene
Experimental: Group 2
Subjects at least 18 y/o treated with a middle dose of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR-ORF15 gene
Experimental: Group 3
Subjects at least 18 y/o treated with a higher dose of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR-ORF15 gene
Experimental: Group 4
Subjects at least 6 y/o treated with a maximum tolerated dose of rAAV2tYF-GRK1-RPGR study drug determined for Groups 1, 2 and 3.
Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR-ORF15 gene
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Number of participants experiencing adverse events
[ Time Frame: Day 0 - Month 36 ]

2. Number of participants experiencing abnormal clinically relevant hematology/clinical chemistry parameters
[ Time Frame: Day 0 - Month 36 ]

Secondary Outcome Measures:
1. Changes from baseline in visual function by perimetry
[ Time Frame: Day 0 - Month 36 ]

2. Changes from baseline in visual acuity by ETDRS
[ Time Frame: Day 0 - Month 36 ]

3. Changes from baseline in retinal structure by OCT
[ Time Frame: Day 0 - Month 36 ]

4. Changes from baseline in VFQ-25 quality of life questionnaire
[ Time Frame: Day 0 - Month 36 ]

Open or close this module Eligibility
Minimum Age: 6 Years
Maximum Age:
Sex: Male
Gender Based: Yes
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Male subjects with the clinical diagnosis of X-linked retinitis pigmentosa (XLRP) and a documented molecular diagnosis from a CLIA-certified laboratory of mutation within the ORF15 exon of the RPGR gene;
  • Best-corrected visual acuity of 20/50 (65 ETDRS letters) or worse in the study eye for Groups 1-3; and best-corrected visual acuity of 20/32 (75 ETDRS letters) or worse in the study eye for Group 4;
  • Ability to perform tests of visual and retinal function and structure and ability to comply with other research procedures;
  • Detectable remaining vision at the intended bleb region;
  • Good general health based on a complete physical examination and hematology and clinical chemistry studies performed at a pre-treatment evaluation;
  • At least 18 years of age for Groups 1, 2 and 3 and at least 6 years of age for Group 4;
  • Has a parent or caregiver able to follow study instructions, comply with the protocol and attend study visits with the subject as required;
  • Signed informed consent and assent (if necessary) obtained before screening.

Exclusion Criteria:

  • Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or increase the risk of surgical complications (for example, glaucoma, corneal or lenticular opacities, diabetic retinopathy, retinal vasculitis);
  • Complicating systemic diseases (such as medical conditions causing immunosuppression or known sensitivity or allergy to medications planned for use in the peri-operative period) that would preclude the gene transfer or ocular surgery;
  • Use of anti-coagulant agents that may alter coagulation within 7 days prior to study agent administration;
  • Use of systemic corticosteroids or other immunosuppressive medications within 3 months prior to enrollment;
  • Males who are unwilling to use barrier contraception for 3 months following agent administration;
  • Any other condition that would prevent a subject from completing follow-up examinations during the course of the study;
  • Any other condition that, in the opinion of the investigator, makes the subject unsuitable for the study;
  • Current, or recent (the longer of 90 days or 10 half-lives of the drug) participation, in any other research protocol involving investigational agents or therapies;
  • Previous receipt of any AAV gene therapy product;
  • Study personnel or family members of the study personnel;
  • Monocular or having BCVA less than 20/800 in the fellow eye.
Open or close this module Contacts/Locations
Central Contact Person: Jill Dolgin, PharmD
Email: advocacy@agtc.com
Study Officials: Matt Feinsod, MD
Study Director
Applied Genetics Technologies Corporation
Locations: United States, North Carolina
Duke Eye Center
Durham, North Carolina, United States, 27701
United States, Oregon
Casey Eye Institute, Oregon Health and Sciences University
Portland, Oregon, United States, 97239
United States, Pennsylvania
University of Pennsylvania Scheie Eye Institute
Philadelphia, Pennsylvania, United States, 19104
Wills Eye Hospital
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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