Actively Recruiting
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Led by Kamau Therapeutics · Updated on 2026-03-11
15
Participants Needed
4
Research Sites
371 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 12 and 40 years
- Diagnosed with severe Sickle Cell Disease
- Experienced at least one of the following despite supportive care: 4 or more severe vaso-occlusive crises in the past 2 years, or 2 or more episodes of acute chest syndrome in the past 2 years with at least one in the past year
- Lansky or Karnofsky performance status score of 80 or higher
You will not qualify if you...
- Having an available 10/10 HLA-matched sibling donor
- Prior hematopoietic stem cell transplant or gene therapy
- History or presence of malignancy, myeloproliferative disorder, significant coagulation disorder, or immunodeficiency
- Active and significant bacterial, viral, fungal, or parasitic infection
- Pregnancy or breastfeeding in postpartum females
- Presence of chromosomal abnormality or mutation that may increase risk for myelodysplastic syndrome or acute myeloid leukemia as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
Actively Recruiting
3
Washington University
St Louis, Missouri, United States, 63110
Actively Recruiting
4
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
Research Team
R
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CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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