NCT04819841 · Kamau Therapeutics
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
(Restore)
What this study is about
This study is a first-in-human, single-treatment group$1, where both patients and doctors know the treatment given 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 effectiveness and how the drug affects the body data.
View original scientific description
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.
Interventions
GENETIC
nula-cel Drug Product
nula-cel is administered via IV infusion following a myeloablative conditioning regimen
Primary outcome measures
Proportion of patients who reach neutrophil engraftment
Time frame: 42 days post-infusion
Incidence rate of treatment-related mortality
Time frame: 100 days post-infusion
Incidence rate of treatment-related mortality
Time frame: 12 months post-infusion
Overall survival
Time frame: 24 months post-infusion
Frequency and severity of AEs/SAEs
Time frame: 24 months post-infusion
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Los Angeles, California
- Palo Alto, California
- St Louis, Missouri
- New York, New York
- Columbus, Ohio
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 2, 2026 · Source of record for eligibility and locations