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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

Status: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 07 Nov 2019

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At a glance

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

    • 07 Nov 2019 According to a Rocket Pharmaceuticals media release, additional Phase 1 Process B data are expected in 1H 2020.
    • 06 Nov 2019 According to a Rocket Pharmaceuticals media release, data from this trial will be presented at the upcoming 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, Florida.
    • 07 Aug 2019 According to a Rocket Pharmaceuticals media release, preliminary data from this trial will be presented in the fourth quarter 2019.
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