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

Trial Profile

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

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 01 Aug 2017

At a glance

  • Drugs SPK 7001 (Primary)
  • Indications Choroideremia
  • Focus Adverse reactions
  • Sponsors Spark Therapeutics
  • Most Recent Events

    • 27 Jul 2017 Status changed from recruiting to active, no longer recruiting.
    • 09 May 2017 According to a Spark Therapeutics media release, interim safety results (n=10) from patients with later stage disease in this trial were announced.
    • 03 May 2017 According to a Spark Therapeutics media release, data from this trial will be presented at Association for Research in Vision and Ophthalmology (ARVO) 2017 Annual Meeting.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top