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Gene Correction for Severe Sickle Cell Disease

This study is testing a treatment called nula-cel for people with severe Sickle Cell Disease. Nula-cel is a gene correction treatment given through an IV (intravenous) infusion after a special conditioning treatment. The study aims to see how safe nula-cel is and if it shows early signs of working. You might be able to join if you are between 12 and 40 years old and have severe Sickle Cell Disease, meaning you've had certain serious events like frequent pain crises or acute chest syndrome. The study will look at how many patients have healthy blood cell growth (neutrophil engraftment) and the number of treatment-related deaths. The study plans to enroll about 15 participants, but its current recruitment status is unclear.

Study design
This is a Phase I/II study, meaning it's an early-stage study looking at safety and initial effectiveness. It's a single-arm, open-label study, so all participants receive the same treatment, and everyone knows what treatment is being given. About 15 participants are planned for enrollment.
What's involved
Participants will receive nula-cel through an IV infusion after a conditioning regimen. The record does not specify the number of visits or the total duration of active participation.
Compensation
Not stated in the trial record.
Follow-up
The study will track participants for treatment-related mortality for up to 12 months after the infusion.

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NCT04819841

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

Recruiting
PHASE1Ages 12–50InterventionalTreatment
Kamau Therapeutics
~15 participants
Updated 2026-07-02 on ClinicalTrials.gov
What's tested:nula-cel Drug Product

At a glance

Recruiting sites
6 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Proportion of patients who reach neutrophil engraftment
Measured over 42 days post-infusion
+4 more outcomes measured

Conditions

Sickle Cell Disease

Explore related trials

Sickle Cell Disease

Where it's being run

6 sites across 4 states
California2
New York2
Missouri1
Ohio1

Study leadership

  • Matthew Porteus, MD, PhD · STUDY_DIRECTOR · Kamau Therapeutics

Who to contact

Restore Clinical Study Support
[email protected]
650-442-2283
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

≥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

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

What this trial measures

  • Proportion of patients who reach neutrophil engraftment42 days post-infusion
  • Incidence rate of treatment-related mortality100 days post-infusion
  • Incidence rate of treatment-related mortality12 months post-infusion
  • Overall survival24 months post-infusion
  • Frequency and severity of AEs/SAEs24 months post-infusion