Gene correction therapy for severe sickle cell disease
Official title: A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
Run by Kamau Therapeutics · NCT04819841 · Data updated 2026-11-12
This study is testing a new gene therapy called nula-cel to see if it can safely improve the health of young people with severe sickle cell disease.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 12 Years to 50 Years |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | Kamau Therapeutics Industry-sponsored |
| Locations | 6 sites (Los Angeles, California and 5 other locations) |
| Status | Recruiting |
| Started | November 15, 2021 |
| Main results expected | December 31, 2026 |
| Study ends | December 31, 2028 |
| Registry record updated | July 2, 2026 |
| Trial ID | NCT04819841 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals aged 12 to 40 with severe sickle cell disease who have experienced recurrent severe vaso-occlusive crises or acute chest syndrome despite appropriate care.
✕Probably not a fit
Patients with a 10/10 HLA-matched sibling donor or those who have previously undergone hematopoietic stem cell transplantation or gene therapy may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study evaluates the safety and preliminary efficacy of nula-cel, a gene correction therapy, in patients with severe sickle cell disease (SCD). Participants aged 12 to 40 will receive the treatment through an IV infusion after undergoing myeloablative conditioning in an autologous hematopoietic stem cell transplant setting. The study aims to gather pharmacodynamic data and assess the overall impact of the therapy on the participants' health.
Why it matters
Potential benefit: If successful, this therapy could significantly improve the quality of life and health outcomes for patients suffering from severe sickle cell disease.
How similar studies have gone: Other studies utilizing gene therapy approaches for sickle cell disease have shown promising results, indicating potential for success in this novel application.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
-
Children's Hospital Los Angeles
Los Angeles, California, United States Recruiting -
Lucile Packard Children's Hospital
Palo Alto, California, United States Recruiting -
Washington University
St Louis, Missouri, United States Recruiting -
Columbia University Irving Medical Center
New York, New York, United States Recruiting -
Memorial Sloan Kettering
New York, New York, United States Recruiting -
Nationwide Children's Hospital
Columbus, Ohio, United States Recruiting
Study contacts
- Study coordinator: Restore Clinical Study Support
- Email: RestoreStudySupport@kamautx.com
- Phone: 650-442-2283
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Hematopoietic stem cell editing to treat sickle cell disease
This study tests whether a patient’s own blood stem cells can be edited to remove the sickle cell triggering gene and then returned to the patient.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.