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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


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

See also
  Status Clinical Trial Phase
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Active, not recruiting NCT00427180 - IRIS PILOT - Extended Pilot Study With a Retinal Implant System N/A
Completed NCT04750785 - A Study to Assess Choroideremia (CHM) Health Outcomes