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Gene Therapy Trial Report

Summary

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


NCTID NCT02341807 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Choroideremia
Disease Ontology Term DOID:9821
Compound Name SPK-7001
Compound Description AAV2-hCHM
Sponsor Spark Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 15 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant CHM
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 5E10 vg/eye (n=5)
Dose 2 1E11 vg/eye (n=10)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2015-01-12
Completion Date 2022-10-12
Last Update 2024-01-25

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility 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 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Roche acquired Spark in December 2019, announced they were halting this program in the 2021 annual report

Resources/Links