Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Mark Walters, MD
Summary
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).
Description
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified CD34+ HSPCs in subjects with severe SCD. The primary endpoint of the trial will determine the safety of CRISPR\_SCD001 through a 3+3 design with staggered enrollment and a pause in enrollment for safety review after each of the first 3 patients has had drug product infused. After safety is assessed in the 3rd patient, enrollment of the next 3 patients will not be staggered. The first six subjects will be adults. If CRISPR\_SCD001 is determined to be safe in the first six subjects, th…
Eligibility
- Age range
- 12–35 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Male or female 12.00 - 34.99 years of age (at time of consent) who have one or more of the following: 1. History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea); ACS is defined as a new radiodensity on chest imaging accompanied by fever and/or respiratory symptoms, treated in the hospital with supplemental oxygen at a minimum. 2. History of at least 4 severe vaso-occlusive pain events in the 2-year period preceding enrollmen…
Interventions
- DrugCRISPR_SCD001
CRISPR\_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan.
Locations (2)
- University of California, Los AngelesLos Angeles, California
- UCSF Benioff Children's HospitalOakland, California