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Trial record 1 of 2 for:    agtc | Retinitis Pigmentosa
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Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects 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: NCT03316560
Recruitment Status : Recruiting
First Posted : October 20, 2017
Last Update Posted : December 11, 2019
Sponsor:
Information provided by (Responsible Party):
Applied Genetic Technologies Corp

Study Description
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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. Approximately 30 participants will be enrolled, and 5 dose levels will be evaluated in a dose-escalation format.

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Biological: rAAV2tYF-GRK1-RPGR Phase 1 Phase 2

Detailed Description:

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

Approximately 30 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). 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 Groups 4 and 7, 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.

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Mutations
Actual Study Start Date : April 16, 2018
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : March 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Group 1
Subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Experimental: Group 2
Subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Experimental: Group 3
Subjects at least 18 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Experimental: Group 4
Subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Experimental: Group 5
Subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Experimental: Group 6
Subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Experimental: Group 7
Subjects at least 6 y/o treated with a maximum tolerated dose of rAAV2tYF-GRK1-RPGR study drug determined by Groups 1-6.
 Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene


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

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male subjects with a documented RPGR mutation within exons 1-14 and/or ORF15 from a CLIA-certified laboratory ;
  • Clinical diagnosis of X-linked retinitis pigmentosa (XLRP);
  • Best-corrected visual acuity not better than 78 ETDRS letters (20/32) in the study eye;
  • 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 1-3, 5 and 6 and at least 6 years of age for Groups 4 and 7;
  • 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;
  • Subjects 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.
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): NCT03316560


Contacts
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Contact: Jill Dolgin, PharmD 833-770-2862 advocacy@agtc.com

Locations
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United States, Colorado
Colorado Retinal Associates Not yet recruiting
Golden, Colorado, United States, 80401
United States, Massachusetts
Boston Children's Hospital Not yet recruiting
Boston, Massachusetts, United States, 02115
United States, New York
Columbia University Recruiting
New York, New York, United States, 10032
United States, North Carolina
Duke Eye Center Recruiting
Durham, North Carolina, United States, 27701
United States, Ohio
Cincinnati Eye Institute Recruiting
Cincinnati, Ohio, United States, 45242
Cleveland Clinic Foundation Not yet recruiting
Cleveland, Ohio, United States, 44195
United States, Oregon
Casey Eye Institute, Oregon Health and Sciences University Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Wills Eye Hospital Not yet recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
Retina Foundation of the Southwest Recruiting
Dallas, Texas, United States, 75231
Sponsors and Collaborators
Applied Genetic Technologies Corp
Investigators
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Study Director: Theresa Heah, MD Applied Genetics Technologies Corporation
More Information
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Responsible Party: Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier: NCT03316560    
Other Study ID Numbers: AGTC-RPGR-001
First Posted: October 20, 2017    Key Record Dates
Last Update Posted: December 11, 2019
Last Verified: May 2019
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
gene therapy
AAV
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