NCT04819841RecruitingPhase 1/2
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat 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
Source: ClinicalTrials.gov · Last updated 2 Jul 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- First submitted
- 24 Mar 2021
- Start date
- 15 Nov 2021
- Primary completion date
- 31 Dec 2026
- Lead sponsor
- Kamau Therapeutics
- Diseases
- Sickle cell disease
- NCT number
- NCT04819841
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 15
- From 15 Nov 2021 to the primary completion date, 31 Dec 2026
- 6 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior HSCT or gene therapy
- Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
Other illness
- Clinically significant and active bacterial, viral, fungal or parasitic infection
Other
- Available 10/10 HLA-matched sibling donor
- 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
Who the study is looking for
- ≥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
Age in the registry: 12 years to 50 years · Sex: All · Does not accept healthy volunteers
Sites (6)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Nationwide Children's Hospital Coordinator of this site? Confirm its status | Columbus | United States | Recruiting |
| Children's Hospital Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Recruiting |
| Memorial Sloan Kettering Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Columbia University Irving Medical Center Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Lucile Packard Children's Hospital Coordinator of this site? Confirm its status | Palo Alto | United States | Recruiting |
| Washington University Coordinator of this site? Confirm its status | St Louis | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.