Recruiting
Phase 1
Phase 2

Gene Correction

Sponsor:

Kamau Therapeutics

Code:

NCT04819841

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 12 - 50

Healthy Volunteers: Not accepted

Interventions

nula-cel Drug Product

Study Details

Brief 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.

Conditions

Sickle Cell Disease

Study ID

NCT04819841

Start date

Nov 15, 2021

Status verified date

Jun, 2026

Completion date

Dec 31, 2028

Anticipated

Primary completion date

Dec 31, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 50

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

15 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: nula-cel Drug Product

nula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.

Interventions

nula-cel Drug Product

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

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

Restore Clinical Study Support

650-442-2283RestoreStudySupport@kamautx.com

Locations

Children's Hospital Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

Bridget Bailey

323-361-7382brbailey@chla.usc.edu

Principal Investigator:

Ashley Gray, MD

Lucile Packard Children's Hospital

Recruiting

Palo Alto, California, United States, 94304

Contacts

Stanford Intake Team

scgt_clinical_trials_office@lists.stanford.edu

Kat Joseph, BSN, RN

650-725-9032kjoseph3@stanford.edu

Principal Investigator:

May Chien, MD

Washington University

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Maggie Nash

314-273-5936nashm@wustl.edu

Principal Investigator:

John F Dipersio, MD, PhD

Columbia University Irving Medical Center

Recruiting

New York, New York, United States, 10032

Contacts

Camila Barros

212-342-3884cb4149@cumc.columbia.edu

Elizabeth Shelton

eas2304@cumc.columbia.edu

Principal Investigator:

Marcus Mapara, MD

Memorial Sloan Kettering

Recruiting

New York, New York, United States, 10065

Contacts

Maria Paes Pena

paespem@mskcc.org

Principal Investigator:

Jaap Jan Boelens, MD, PhD

Nationwide Children's Hospital

Recruiting

Columbus, Ohio, United States, 43205

Contacts

Dalena Sanderson

Dalena.Sanderson@nationwidechildrens.org

Lauren Rayman

lauren.rayman2@nationwidechildrens.org

More Information

Sponsor

Kamau Therapeutics

Last update posted

Jul 2, 2026

Last verified

Jun, 2026

Keywords

  • sickle cell disease
  • sickle cell anemia
  • gene correction
  • gene therapy
  • CRISPR

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-08. This information was provided to ClinicalTrials.gov by Kamau Therapeutics on 2026-07-02. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.