This is a first-in-human, single-arm, open-label Phase 1/2 study of nula-cel in approximately 15 participants diagnosed with severe sickle cell disease (SCD). The approach corrects the HbS gene to HbA in the patient's own CD34+ hematopoietic stem cells; the primary objective is safety, with preliminary efficacy and pharmacodynamic data also collected. Eligibility requires an age of 12 to 40 years and severe disease, defined by events such as at least 4 severe vaso-occlusive crises in the preceding 2 years or at least 2 episodes of acute chest syndrome in the prior 2 years despite appropriate supportive care.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * ≥12 to ≤ 40 years * Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures: * recurrent severe VOC (≥ 4 episodes in the preceding 2 years) * ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year) * Lansky/Karnofsky performance status of ≥ 80 Exclusion Criteria: * Available 10/10 HLA-matched sibling donor * Prior HSCT or gene therapy * Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder * Clinically significant and active bacterial, viral, fungal or parasitic infection * Pregnancy or breastfeeding in a postpartum female * Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment