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A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A

Trial Profile

A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A

Recruiting
Phase of Trial: Phase I

Latest Information Update: 31 Jan 2019

At a glance

  • Drugs RP L102 (Primary)
  • Indications Fanconi's anaemia
  • Focus Adverse reactions
  • Sponsors Rocket Pharmaceuticals
  • Most Recent Events

    • 18 Jan 2019 Status changed from planning to recruiting.
    • 25 Nov 2018 New trial record
    • 07 Nov 2018 According to a Rocket Pharmaceuticals media release, the company looks forward to engaging with regulatory authorities on a final registration path in the second half of 2019 after initial patients have been treated in this trial.
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