Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Recruiting now · Phase 1/Phase 2
Conditions studied: Sickle Cell Disease
In brief
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.
Key facts
- Study ID
- NCT04819841
- Run by
- Kamau Therapeutics
- People needed
- 15
- Starts
- 2021-11-15
- Expected to finish
- 2028-12-31
- Last updated by the study team
- 2026-07-02
Who can join
Age: 12 and older, up to 50. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- ≥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
You may not qualify if…
- 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
Where it is running
- Children's Hospital Los Angeles — Los Angeles, California, United States (enrolling)
- Lucile Packard Children's Hospital — Palo Alto, California, United States (enrolling)
- Washington University — St Louis, Missouri, United States (enrolling)
- Columbia University Irving Medical Center — New York, New York, United States (enrolling)
- Memorial Sloan Kettering — New York, New York, United States (enrolling)
- Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
Full record on ClinicalTrials.gov
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