NCT02341807

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

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Spark Therapeutics, Inc. · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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).

Conditions

• Choroideremia
• CHM (Choroideremia) Gene Mutations

Interventions

Type	Name	Description
BIOLOGICAL	AAV2-hCHM	Comparison of different dosages of AAV2-hCHM

Timeline

• Start date: 2015-01-15
• Primary completion: 2022-10-12
• Completion: 2022-10-12
• First posted: 2015-01-19
• Last updated: 2024-01-25
• Results posted: 2023-11-02

Locations

3 sites across 1 country: United States