A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Last updated: October 30, 2024
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Active - Not Recruiting

Phase

2/3

Condition

Sickle Cell Disease

Hematological Disorders

Red Blood Cell Disorders

Treatment

CTX001

Clinical Study ID

NCT03745287
CTX001-121
  • Ages 12-35
  • All Genders

Study Summary

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Diagnosis of severe sickle cell disease as defined by:

  • Documented severe sickle cell disease genotype

  • History of at least two severe vaso-occlusive crisis events per year for theprevious two years prior to enrollment

  • Eligible for autologous stem cell transplant as per investigators judgment

Exclusion

Key Exclusion Criteria:

  • An available 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: 63
Treatment Group(s): 1
Primary Treatment: CTX001
Phase: 2/3
Study Start date:
November 27, 2018
Estimated Completion Date:
July 31, 2025

Connect with a study center

  • Hopital Universitaire des Enfants Reine Fabiola (HUDERF)

    Brussels,
    Belgium

    Site Not Available

  • The Hospital for Sick Children

    Toronto,
    Canada

    Site Not Available

  • Hopital Necker Enfants Malades

    Paris,
    France

    Site Not Available

  • University Hospital Duesseldorf

    Dusseldorf,
    Germany

    Site Not Available

  • Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation

    Regensburg,
    Germany

    Site Not Available

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

    Rome,
    Italy

    Site Not Available

  • Imperial College Healthcare NHS Trust, Hammersmith Hospital

    London,
    United Kingdom

    Site Not Available

  • Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building

    London,
    United Kingdom

    Site Not Available

  • Lucile Packard Children's Hospital of Stanford University

    Palo Alto, California 94304
    United States

    Site Not Available

  • Lucille Packard Children's Hospital of Stanford University

    Palo Alto, California 94304
    United States

    Site Not Available

  • Ann & Robert Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Site Not Available

  • University of Illinois at Chicago Hospitals and Health Systems

    Chicago, Illinois 60612
    United States

    Site Not Available

  • Columbia University Medical Center (21+ years)

    New York, New York 10032
    United States

    Site Not Available

  • Columbia University Medical Center (≤21 years)

    New York, New York 10032
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • 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

  • Methodist Children's Hospital/Texas Transplant Institute

    San Antonio, Texas 78229
    United States

    Site Not Available

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