Gene correction therapy for severe sickle cell disease
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
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 |
| Enrollment | 15 (estimated) |
| Ages | 12 Years to 40 Years |
| Sex | All |
| Sponsor | Kamau Therapeutics Industry-sponsored |
| Locations | 4 sites (Los Angeles, California and 3 other locations) |
| Trial ID | NCT04819841 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: Other studies utilizing gene therapy approaches for sickle cell disease have shown promising results, indicating potential for success in this novel application.
Eligibility criteria
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 this trial is running
Los Angeles, California and 3 other locations
- 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)
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.