Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Completed · Phase 1/Phase 2

Conditions studied: Choroideremia, CHM (Choroideremia) Gene Mutations

In brief

This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.

Key facts

Study ID

NCT02341807

Run by

Spark Therapeutics, Inc.

People needed

15

Starts

2015-01-15

Expected to finish

2022-10-12

Last updated by the study team

2024-01-25

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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°

You may not qualify if…

• 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 Early Treatment of Diabetic Retinopathy Study (ETDRS) testing in the eye to be injected
• Presence of disease which may preclude the participant 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.

Where it is running

• Massachusetts Eye and Ear Infirmary — Boston, Massachusetts, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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