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Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene 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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02341807
Recruitment Status : Completed
First Posted : January 19, 2015
Last Update Posted : October 31, 2022
Sponsor:
Information provided by (Responsible Party):
Spark Therapeutics

Study Description
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Brief Summary:
This clinical study evaluates the safety and tolerability of AAV2-hCHM in subjects with Choroideremia gene mutations. Two dose groups will be evaluated.

Condition or disease Intervention/treatment Phase
Choroideremia CHM (Choroideremia) Gene Mutations Biological: AAV2-hCHM Phase 1 Phase 2

Detailed Description:
The primary objective is to evaluate the safety and tolerability of subretinal administration of AAV2-hCHM, in an inter-subject group dose escalation in individuals with choroideremia, based on a comprehensive clinical monitoring plan. The secondary objectives are to define the dose of AAV2-hCHM required to achieve stable, or improved, visual function/functional vision and to assess development of immune responses to AAV2 and REP-1.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the Retina
Actual Study Start Date : January 2015
Actual Primary Completion Date : October 12, 2022
Actual Study Completion Date : October 12, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Dose Group 1
Single, unilateral administration of a single low dose range of AAV2-hCHM.
 Biological: AAV2-hCHM
Comparison of different dosages of AAV2-hCHM
Experimental: Dose Group 2
Single, unilateral administration of a single high dose range of AAV2-hCHM.
 Biological: AAV2-hCHM
Comparison of different dosages of AAV2-hCHM


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability (assessed by physical exam, vital signs, laboratory changes over time, and adverse events) [ Time Frame: 5 years ]
    Safety and tolerability of a single dose of AAV2-hCHM will be assessed by physical exam, vital signs, laboratory changes over time, and adverse events.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male at least 18 years of age diagnosed with CHM gene mutation
  • Central visual field (VF) < 30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected
  • Any evidence of functioning outer retinal cells within the central 10°

Exclusion Criteria:

  • Previous history of ocular inflammatory disease (uveitis)
  • Prior intraocular surgery within six months
  • Participation in a previous gene therapy research trial within one year of enrollment or participation in any other ocular gene therapy trial
  • Participation in a clinical study with an investigational drug in the past six months
  • Grossly asymmetrical disease, or other eye morbidity, which may render the contralateral eye ineffective as a control
  • Visual acuity < 20/200 on standard ETDRS testing in the eye to be injected
  • Presence of disease which may preclude the subject from participation in this trial
  • Use of medications known to be neuroprotective or retino-toxic that could potentially interfere with the disease process and/or cause ocular adverse events; individuals who discontinue use of these compounds for 6 months may become eligible
  • Identification by the investigator as being unable or unwilling to perform / be compliant with study procedures.
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): NCT02341807


Locations
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United States, Massachusetts
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States, 02114
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19014
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Spark Therapeutics
Investigators
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Study Director: Clinical Director Spark Therapeutics
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT02341807    
Other Study ID Numbers: AAV2-hCHM-101
First Posted: January 19, 2015    Key Record Dates
Last Update Posted: October 31, 2022
Last Verified: October 2022
Keywords provided by Spark Therapeutics:
Choroideremia
AAV
Gene therapy
 CHM
Adeno-associated virus
Adeno-associated viral vector
Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
 Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked