Condition: Sickle Cell Disease · Sponsor: Mark Walters, MD
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).
This description comes directly from the study's public registry record.
Mark Walters, MD · (510) 428-3374 · Mark.Walters@ucsf.edu
Christina Chun, MPH · (415) 502-2558 · Christina.Chun@ucsf.edu
Always discuss trial participation with your own doctor first.
| University of California, Los Angeles | Los Angeles, California, United States | Recruiting |
| UCSF Benioff Children's Hospital | Oakland, California, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT04774536