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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-01-15

Study Completion Date

2022-10-12

Brief Summary

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

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Detailed Description

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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 adeno-associated virus vector, serotype 2 (AAV2) and Rab escort protein 1 (REP-1).

Conditions

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Choroideremia CHM (Choroideremia) Gene Mutations

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1: AAV2-hCHM Dose 1

Single, unilateral subretinal administration of a single low dose range of AAV2-hCHM.

Group Type EXPERIMENTAL

AAV2-hCHM

Intervention Type BIOLOGICAL

Comparison of different dosages of AAV2-hCHM

Cohort 2: AAV2-hCHM Dose 2

Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM.

Group Type EXPERIMENTAL

AAV2-hCHM

Intervention Type BIOLOGICAL

Comparison of different dosages of AAV2-hCHM

Cohort 3 (Expansion Cohort): AAV2-hCHM Dose 2

Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM.

Group Type EXPERIMENTAL

AAV2-hCHM

Intervention Type BIOLOGICAL

Comparison of different dosages of AAV2-hCHM

Interventions

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AAV2-hCHM

Comparison of different dosages of AAV2-hCHM

Intervention Type BIOLOGICAL

Eligibility Criteria

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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 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.

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No