CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
Terminated · Phase 1 · NCT04091737
Conditions: Anemia, Sickle Cell
At a glance
- Research payments reported, 2025
- $1,786
- U.S. investigators named
- 0
- Sites paid
- 1
- Planned or actual enrollment
- 1
About the trial
- Sponsor
- CSL Behring
- Tested
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 (biological)
- Dates
- Started 2019-10-02; completion 2021-05-05
- Results posted
- Not yet
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.
From ClinicalTrials.gov, last updated 2021-06-18. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Csl Behring reported $1,786 in research payments for this study in 2025 (1 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
Sites paid to run it
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.