Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Recruiting · NCT04819841 · Interventional (participants receive a specific treatment) · Lead sponsor: Kamau Therapeutics
View the official record on ClinicalTrials.gov →Interventions studied
nula-cel Drug Product
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.
Who can take part
Age range
12 Years to 50 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 1, Phase 2
Study type
Interventional (participants receive a specific treatment)
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 it is running
6 locations listed across 4 US states.
- Children's Hospital Los Angeles - Los Angeles, California, United States
- Lucile Packard Children's Hospital - Palo Alto, California, United States
- Washington University - St Louis, Missouri, United States
- Columbia University Irving Medical Center - New York, New York, United States
- Memorial Sloan Kettering - New York, New York, United States
- Nationwide Children's Hospital - Columbus, Ohio, United States