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This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)
Full description
This clinical trial is a single-arm, single-dose, single center, open-label study without dose escalation. The primary objective is to explore the safety of the study drug in SCD. Myeloablative conditioning and administration for the remaining subjects can only be started after the first subject completes dosing and safety observation and assessment.
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Inclusion and exclusion criteria
Inclusion Criteria:
Subjects must meet all the following inclusion criteria to be eligible for enrolment into the study:
Exclusion Criteria
Subjects meeting any of the following criteria are not eligible for enrolment in the study:
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Interventional model
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1 participants in 1 patient group
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Central trial contact
Jun shi, PhD
Data sourced from clinicaltrials.gov
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