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RecruitingPhase 1/Phase 2

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

Kamau Therapeutics · NCT04819841

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 12–50 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
15
US sites recruiting
6
Study start
November 15, 2021
Record last updated
July 2, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: nula-cel Drug Product

Conditions: Sickle Cell Disease

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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

Recruiting US locations

  • Children's Hospital Los Angeles

    Los Angeles, CA 90027

    Site contact: Bridget Bailey · 323-361-7382 · brbailey@chla.usc.edu

  • Lucile Packard Children's Hospital

    Palo Alto, CA 94304

    Site contact: Stanford Intake Team · scgt_clinical_trials_office@lists.stanford.edu

  • Washington University

    St Louis, MO 63110

    Site contact: Maggie Nash · 314-273-5936 · nashm@wustl.edu

  • Columbia University Irving Medical Center

    New York, NY 10032

    Site contact: Camila Barros · 212-342-3884 · cb4149@cumc.columbia.edu

  • Memorial Sloan Kettering

    New York, NY 10065

    Site contact: Maria Paes Pena · paespem@mskcc.org

  • Nationwide Children's Hospital

    Columbus, OH 43205

    Site contact: Dalena Sanderson · Dalena.Sanderson@nationwidechildrens.org

Study contacts (from the official record)

  • Restore Clinical Study Support · 650-442-2283 · RestoreStudySupport@kamautx.com
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Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS… (NCT04819841) | Bored Studies