Status:
RECRUITING
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Lead Sponsor:
Kamau Therapeutics
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
12-40 years
Phase:
PHASE1
PHASE2
Brief Summary
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 sa...
Detailed Description
Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting.
Eligibility Criteria
Inclusion
- ≥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
- 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
Key Trial Info
Start Date :
November 15 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2028
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT04819841
Start Date
November 15 2021
End Date
December 31 2028
Last Update
November 21 2025
Active Locations (4)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
3
Washington University
St Louis, Missouri, United States, 63110
4
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205