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Clinical Trial Report

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
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
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 5E10 vg/eye
Dose 2 1E11 vg/eye
Dose 3
Dose 4
Dose 5

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

Locations

No.of Trial Sites 3
Locations United States

Regulatory Information

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

Resources/Links

Clinical Publications
News and Press Releases
Preclinical Publications
Protocol