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Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia

This study is currently recruiting participants.
See Contacts and Locations
Verified June 2017 by Applied Genetic Technologies Corp
Sponsor:
Information provided by (Responsible Party):
Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier:
NCT02935517
First received: October 13, 2016
Last updated: June 27, 2017
Last verified: June 2017
  Purpose
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

Condition Intervention Phase
Achromatopsia Biological: AGTC-402 Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC 402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients With Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene

Resource links provided by NLM:


Further study details as provided by Applied Genetic Technologies Corp:

Primary Outcome Measures:
  • Adverse events [ Time Frame: 1 year ]
    Proportion of participants experiencing grade 3 or greater adverse events


Secondary Outcome Measures:
  • Visual acuity [ Time Frame: 1 year ]
    Changes in best corrected visual acuity compared to pre-treatment

  • Light aversion [ Time Frame: 1 year ]
    Changes in light discomfort testing compared to pre-treatment

  • Color vision [ Time Frame: 1 year ]
    Changes in color vision testing compared to pre-treatment


Estimated Enrollment: 24
Actual Study Start Date: May 1, 2017
Estimated Study Completion Date: June 2023
Estimated Primary Completion Date: June 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lower dose
AGTC-402 will be administered at the lowest of three planned dose levels.
Biological: AGTC-402
AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene.
Other Name: rAAV2tYF-PR1.7-hCNGA3
Experimental: Middle dose
AGTC-402 will be administered at the middle of three planned dose levels.
Biological: AGTC-402
AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene.
Other Name: rAAV2tYF-PR1.7-hCNGA3
Experimental: Higher dose
AGTC-402 will be administered at the highest of three planned dose levels.
Biological: AGTC-402
AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene.
Other Name: rAAV2tYF-PR1.7-hCNGA3
Experimental: Maximum tolerated dose
AGTC-402 will be administered at the maximum tolerated dose identified from Groups 1, 2 and 3.
Biological: AGTC-402
AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene.
Other Name: rAAV2tYF-PR1.7-hCNGA3

Detailed Description:

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

Subjects will be enrolled sequentially in four groups. Subjects in Groups 1, 2 and 3 will be at least 18 years of age and will receive a lower, middle or higher dose of study agent. Subjects in Group 4 will be at least 6 years of age and will receive the maximum tolerated dose identified in Groups 1, 2 and 3.

Safety will be monitored by evaluation of ocular and non ocular adverse events and hematology and clinical chemistry parameters. Efficacy parameters will include visual acuity, light discomfort testing, color vision, static visual field, ERG, adaptive optics retinal imaging and OCT.

  Eligibility

Ages Eligible for Study:   6 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Retinal disease consistent with a diagnosis of achromatopsia and documented mutations in both alleles of the CNGA3 gene;
  2. At least 18 years of age for Groups 1, 2 and 3 and at least 6 years of age for Group 4;
  3. Able to perform tests of visual and retinal function;
  4. Visual acuity in the study eye not better than 55 ETDRS letters (Snellen equivalent 20/80) based on the average of two examinations at the baseline visit;
  5. Acceptable laboratory parameters;
  6. For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.

Exclusion Criteria:

  1. Presence of myopia which, in the opinion of Investigator, may create increased surgical risk for the subject in the study eye;
  2. Evidence of degenerative myopia in the study eye;
  3. Pre-existing eye conditions that would contribute to vision loss in either eye or increase the risk of subretinal injection in the study eye.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02935517

Contacts
Contact: Jill Dolgin, PharmD advocacy@agtc.com

Locations
United States, Florida
VitreoRetinal Associates Recruiting
Gainesville, Florida, United States, 32607
Bascom Palmer Eye Institute Recruiting
Miami, Florida, United States, 33136
United States, Massachusetts
Massachusetts Eye and Ear Infirmary Recruiting
Boston, Massachusetts, United States, 02114
United States, Oregon
Casey Eye Institute, Oregon Health and Sciences University Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
University of Pennsylvania Active, not recruiting
Philadelphia, Pennsylvania, United States, 19104
Israel
Hadassah-Hebrew University Medical Center Not yet recruiting
Jerusalem, Israel, 91120
Sponsors and Collaborators
Applied Genetic Technologies Corp
Investigators
Study Director: Mike Goldstein, MD Applied Genetics Technologies Corporation
  More Information

Additional Information:
Publications:
Responsible Party: Applied Genetic Technologies Corp
ClinicalTrials.gov Identifier: NCT02935517     History of Changes
Other Study ID Numbers: AGTC-CNGA3-002
Study First Received: October 13, 2016
Last Updated: June 27, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
Color vision deficiency
Color vision defects
Vision disorders
Sensation disorders
Neurologic manifestations
Nervous system diseases
Eye diseases
Signs and symptoms

Additional relevant MeSH terms:
Color Vision Defects
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Eye Diseases
Signs and Symptoms

ClinicalTrials.gov processed this record on July 24, 2017