Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Part of paid clinical trials in Los Angeles, California.
- Sponsor
- Kamau Therapeutics
- Study ID
- NCT04819841
- Phase
- PHASE1/PHASE2
- Status
- Recruiting
Conditions
Eligibility Criteria
- Sex
- ALL
- Age
- 12 Years - 40 Years
- Healthy Volunteers
- Not accepted
Interventions
- nula-cel Drug Product — GENETICnula-cel is administered via IV infusion following a myeloablative conditioning regimen
Study Details
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.
Key Dates
- Start date
- Nov 15, 2021
- Status verified
- Mar 2026
- Primary completion
- Dec 31, 2026
- Completion
- Dec 31, 2028
Study Design
- Enrollment
- 15 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: nula-cel Drug Productnula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.
Primary Outcome Measure
Proportion of patients who reach neutrophil engraftment [ Time Frame: 42 days post-infusion ]
Central Contacts
- Restore Clinical Study Support650-442-2283
Locations (4)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | Ashley Gray, MD (PRINCIPAL_INVESTIGATOR) |
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | Stanford Intake Team May Chien, MD (PRINCIPAL_INVESTIGATOR) David Shyr, MD (SUB_INVESTIGATOR) |
| Washington University | St Louis | Missouri | 63110 | John F Dipersio, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Dalena Sanderson Lauren Rayman |
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