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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 : Active, not recruiting
First Posted : January 19, 2015
Last Update Posted : March 12, 2019
Sponsor:
Collaborators:
Children's Hospital of Philadelphia
University of Pennsylvania
Massachusetts Eye and Ear Infirmary
Information provided by (Responsible Party):
Spark Therapeutics

Tracking Information
First Submitted Date  ICMJE January 12, 2015
First Posted Date  ICMJE January 19, 2015
Last Update Posted Date March 12, 2019
Study Start Date  ICMJE January 2015
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 14, 2015)
Safety and tolerability (assessed by physical exam, vital signs, laboratory changes over time, and adverse events) [ Time Frame: 2 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02341807 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Outcome Measures  ICMJE Not Provided
Original Other Outcome Measures  ICMJE Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations
Official Title  ICMJE 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
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.
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 Type  ICMJE Interventional
Study Phase Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Choroideremia
  • CHM (Choroideremia) Gene Mutations
Intervention  ICMJE Biological: AAV2-hCHM
Comparison of different dosages of AAV2-hCHM
Study Arms
  • Experimental: Dose Group 1
    Single, unilateral administration of a single low dose range of AAV2-hCHM.
    Intervention: Biological: AAV2-hCHM
  • Experimental: Dose Group 2
    Single, unilateral administration of a single high dose range of AAV2-hCHM.
    Intervention: Biological: AAV2-hCHM
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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 20, 2016)
15
Original Estimated Enrollment  ICMJE
 (submitted: January 14, 2015)
10
Estimated Study Completion Date October 2019
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender
Sexes Eligible for Study: Male
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02341807
Other Study ID Numbers  ICMJE AAV2-hCHM-101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Spark Therapeutics
Study Sponsor  ICMJE Spark Therapeutics
Collaborators  ICMJE
  • Children's Hospital of Philadelphia
  • University of Pennsylvania
  • Massachusetts Eye and Ear Infirmary
Investigators  ICMJE
Study Director: Clinical Director Spark Therapeutics
PRS Account Spark Therapeutics
Verification Date March 2019

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