Status: Recruiting
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Participants needed: 15
Trial details
Phase: Phase 1, Phase 2Age: 12-50Biological sex: AllType: InterventionalSponsor: Kamau TherapeuticsUpdated: Jul 2, 2026Locations: 6
Eligibility criteria
≥12 to ≤ 40 years [+3]
Available 10/10 HLA-matched sibling donor [+5]