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

Choroideremia Clinical Trial

Official title:
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

Clinical Trial Summary

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

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

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02341807
Study type Interventional
Source Spark Therapeutics
Contact
Status Completed
Phase Phase 1/Phase 2
Start date January 15, 2015
Completion date October 12, 2022
View Details
See also
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