Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
About this study
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.
- Condition
- Sickle Cell Disease
- Tested
- nula-cel Drug Product
- Sponsor
- Kamau Therapeutics
Who can join
- Age
- 12 to 50 years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
Inclusion 8
- Minimum age: 12 years
- Maximum age: 50 years
- Study condition: Sickle Cell Disease
- ≥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 6
- 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
6 sites, 6 recruiting
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Columbia University Irving Medical Center
New York, New York, United States
Memorial Sloan Kettering
New York, New York, United States
Lucile Packard Children's Hospital
Palo Alto, California, United States
Washington University
St Louis, Missouri, United States
Contact
-
Restore Clinical Study Support
650-442-2283 RestoreStudySupport@kamautx.com
Potential match only. Final eligibility is determined by the study team.