A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients With Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene

Trial Profile

A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients With Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 30 Jun 2017

At a glance

  • Drugs AAV CNGB3 (Primary)
  • Indications Colour vision defects
  • Focus Adverse reactions; First in man
  • Sponsors Applied Genetic Technologies Corporation
  • Most Recent Events

    • 10 May 2017 According to an AGTC media release, the company plans to enroll two additional patients in the low dose group who will both be treated by a surgeon who treated one of the earlier patients in this group, such that a total of three patients will be treated by a single surgeon.
    • 08 Nov 2016 According to an AGTC media release, three patients in the low dose group have been enrolled in this trial as of October 2016 and as specified in the protocol, safety data from these three patients will be reviewed by a DSMC before the company enrolls patietns in th middle dose group.
    • 12 Sep 2016 According to an Applied Genetic Technologies Corporation media release, enrollment in the study is slower-than-expected and was due to vendor errors identified during testing of the study agent. The company believes that it has resolved these factors in order to meet its future enrollment goals.
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