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A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B

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Trial Profile

A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 11 Jan 2022

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At a glance

  • Drugs DTX 101 (Primary)
  • Indications Haemophilia B
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors Dimension Therapeutics; Ultragenyx Pharmaceutical

    • Most Recent Events

    • 17 Sep 2019 Planned End Date changed from 1 Feb 2022 to 18 Feb 2022.
    • 17 Sep 2019 Planned primary completion date changed from 1 Feb 2022 to 18 Feb 2022.
    • 08 Aug 2017 According to a Dimension Therapeutics media release, the company will continue to monitor all patients dosed in this trial for up to five years post dosing.

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