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

NCT04819841 · tracked via the Priya Life Science USA tracker

Sponsor

Kamau Therapeutics

Phase

Phase 1/2

Started

2021-11-15

Last updated

2026-07-02

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

nula-cel Drug Product

nula-cel Drug Product: nula-cel is administered via IV infusion following a myeloablative conditioning regimen

Study 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 safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Eligibility

Sex

ALL

Min age

12 Years

Max age

50 Years

Healthy volunteers

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

Primary outcome measure(s)

Proportion of patients who reach neutrophil engraftment — 42 days post-infusion
Incidence rate of treatment-related mortality — 100 days post-infusion
Incidence rate of treatment-related mortality — 12 months post-infusion
Overall survival — 24 months post-infusion
Frequency and severity of AEs/SAEs — 24 months post-infusion

Trial sites (6)

Facility	City	Region	Status
Children's Hospital Los Angeles	Los Angeles	California	Recruiting
Lucile Packard Children's Hospital	Palo Alto	California	Recruiting
Washington University	St Louis	Missouri	Recruiting
Columbia University Irving Medical Center	New York	New York	Recruiting
Memorial Sloan Kettering	New York	New York	Recruiting
Nationwide Children's Hospital	Columbus	Ohio	Recruiting

Other trials for the same condition

A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease Recruiting

Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) Recruiting

Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease Recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Active, not recruiting

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Enrolling by invitation

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04819841 on ClinicalTrials.gov ↗ ← All trials in the USA