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: 07 Nov 2017

At a glance

  • Drugs A002-MeiraGTx (Primary)
  • Indications Colour vision defects
  • Focus Adverse reactions; First in man
  • Sponsors Applied Genetic Technologies Corporation
  • Most Recent Events

    • 07 Nov 2017 According to an Applied Genetic Technologies Corporation media release, the company has found evidence of biological activity at lower vector doses than those originally proposed for the ACHM B3 study and has elected to adjust the dose downward in this dose ranging study.
    • 13 Sep 2017 According to Applied Genetic Technologies Corporation media release, the company has enrolled four patients in the low-dose group and collecting data from this group to present to Data Safety and Monitoring Committee (DSMC) prior to enrollment of patients in the mid-dose group
    • 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.
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