Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Last updated: October 13, 2025
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Active - Recruiting

Phase

3

Condition

Hematological Disorders

Sickle Cell Disease

Red Blood Cell Disorders

Treatment

CTX001

Clinical Study ID

NCT05329649
VX21-CTX001-151
2021-002173-26
  • Ages 2-11
  • All Genders

Study Summary

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Diagnosis of severe SCD as defined by:

  • Documented SCD genotypes

  • History of at least two severe VOCs events per year for the previous two years priorto enrollment

  • Hydroxyurea (HU) failure unless HU intolerant

  • Eligible for autologous stem cell transplant as per investigators judgment

Exclusion

Key Exclusion Criteria:

  • A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor

  • Prior hematopoietic stem cell transplant (HSCT).

  • Clinically significant and active bacterial, viral, fungal, or parasitic infection

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 15
Treatment Group(s): 1
Primary Treatment: CTX001
Phase: 3
Study Start date:
May 02, 2022
Estimated Completion Date:
May 31, 2026

Connect with a study center

  • University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

    Dusseldorf,
    Germany

    Site Not Available

  • University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

    Düsseldorf 2934246,
    Germany

    Active - Recruiting

  • Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS

    Rome,
    Italy

    Site Not Available

  • IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

    Rome 3169070,
    Italy

    Active - Recruiting

  • St Mary's Hospital

    London,
    United Kingdom

    Site Not Available

  • St.Mary's Hospital - Haematology Dept

    London 2643743,
    United Kingdom

    Active - Recruiting

  • Atrium Health Levine Children's Hospital

    Charlotte, North Carolina 28203
    United States

    Site Not Available

  • Levine Children's Hospital - Hematology

    Charlotte 4460243, North Carolina 4482348 28203
    United States

    Active - Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • The Children's Hospital of Philadelphia - Hematology

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Active - Recruiting

  • St. Jude Children's Research Hospital

    Memphis, Tennessee 38105
    United States

    Site Not Available

  • The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers

    Nashville, Tennessee 37203
    United States

    Site Not Available

  • St. Jude Children's Research Hospital

    Memphis 4641239, Tennessee 4662168 38105
    United States

    Active - Recruiting

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    Nashville 4644585, Tennessee 4662168 37203
    United States

    Active - Recruiting

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