Working…
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 8 for:    AGTC

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

Tracking Information
First Submitted Date  ICMJE October 10, 2017
First Posted Date  ICMJE October 20, 2017
Last Update Posted Date December 11, 2019
Actual Study Start Date  ICMJE April 16, 2018
Estimated Primary Completion Date March 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 17, 2017)
  • Number of participants experiencing adverse events [ Time Frame: Day 0 - Month 36 ]
  • Number of participants experiencing abnormal clinically relevant hematology/clinical chemistry parameters [ Time Frame: Day 0 - Month 36 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03316560 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 22, 2019)
  • Changes from baseline in visual function by perimetry [ Time Frame: Day 0 - Month 36 ]
  • Changes from baseline in visual acuity by ETDRS [ Time Frame: Day 0 - Month 36 ]
  • Changes from baseline in retinal structure by imaging [ Time Frame: Day 0 - Month 36 ]
  • Changes from baseline in quality of life questionnaire [ Time Frame: Day 0 - Month 36 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: October 17, 2017)
  • Changes from baseline in visual function by perimetry [ Time Frame: Day 0 - Month 36 ]
  • Changes from baseline in visual acuity by ETDRS [ Time Frame: Day 0 - Month 36 ]
  • Changes from baseline in retinal structure by OCT [ Time Frame: Day 0 - Month 36 ]
  • Changes from baseline in VFQ-25 quality of life questionnaire [ Time Frame: Day 0 - Month 36 ]
Current Other Pre-specified Outcome Measures Not Provided
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 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
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE X-Linked Retinitis Pigmentosa
Intervention  ICMJE Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Study Arms  ICMJE
  • Experimental: Group 1
    Subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
  • Experimental: Group 2
    Subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
  • Experimental: Group 3
    Subjects at least 18 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
  • Experimental: Group 4
    Subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
  • Experimental: Group 5
    Subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
  • Experimental: Group 6
    Subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
  • 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.
    Intervention: Biological: rAAV2tYF-GRK1-RPGR
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 22, 2019)
30
Original Estimated Enrollment  ICMJE
 (submitted: October 17, 2017)
15
Estimated Study Completion Date  ICMJE March 2025
Estimated Primary Completion Date March 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jill Dolgin, PharmD 833-770-2862 advocacy@agtc.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03316560
Other Study ID Numbers  ICMJE AGTC-RPGR-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Applied Genetic Technologies Corp
Study Sponsor  ICMJE Applied Genetic Technologies Corp
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Theresa Heah, MD Applied Genetics Technologies Corporation
PRS Account Applied Genetic Technologies Corp
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP