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A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 (Autologous Enriched CD34+ Cell Fraction That Contains CD34+ Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734) in Adult Subjects With Severe Sickle Cell Disease

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A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 (Autologous Enriched CD34+ Cell Fraction That Contains CD34+ Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734) in Adult Subjects With Severe Sickle Cell Disease

Status: Discontinued
Phase of Trial: Phase I

Latest Information Update: 04 Nov 2021

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At a glance

  • Drugs CSL 200 (Primary) ; Melphalan; Plerixafor
  • Indications Sickle cell anaemia
  • Focus Adverse reactions
  • Sponsors CSL Behring

    • Most Recent Events

    • 14 Jun 2021 Status changed from active, no longer recruiting to discontinued.
    • 03 Feb 2021 Status changed from recruiting to active, no longer recruiting.
    • 01 Jul 2020 Status changed from active, no longer recruiting to recruiting.

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