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Study identifier: NCT04819841 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Condition: Sickle Cell Disease  ·  Sponsor: Kamau Therapeutics

PhasePhase 1/Phase 2
Planned participants15
Who can joinAll sexes, 12 Years to 50 Years
Healthy volunteersNo

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.

This description comes directly from the study's public registry record.

Talk to the study team

Restore Clinical Study Support  ·  650-442-2283  ·  RestoreStudySupport@kamautx.com

Always discuss trial participation with your own doctor first.

Locations (6)

Children's Hospital Los AngelesLos Angeles, California, United StatesRecruiting
Lucile Packard Children's HospitalPalo Alto, California, United StatesRecruiting
Washington UniversitySt Louis, Missouri, United StatesRecruiting
Columbia University Irving Medical CenterNew York, New York, United StatesRecruiting
Memorial Sloan KetteringNew York, New York, United StatesRecruiting
Nationwide Children's HospitalColumbus, Ohio, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT04819841