A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Study point of contact

Medical Information
6173416777
[email protected]

Locations

8 United States sites

1 Belgium site

1 Canada site

1 Germany site

1 Italy site

1 United Kingdom site

Age

12 to 35 Years

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Biological

Compensation

Unknown

About the study

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 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.

participation requirements

– 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 the previous
two years prior to enrollment

– Eligible for autologous stem cell transplant as per investigators judgment

Key

participation restrictions

– 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

Locations

  • Palo Alto, California, United States, Lucille Packard Children's Hospital of Stanford University, 94304 [Recruiting]
  • Chicago, Illinois, United States, University of Illinois at Chicago Hospitals and Health Systems, 60612 [Recruiting]
  • New York, New York, United States, Columbia University Medical Center (21+ years), 10032 [Recruiting]
  • New York, New York, United States, Columbia University Medical Center (≤21 years), 10032 [Recruiting]
  • Philadelphia, Pennsylvania, United States, Children's Hospital of Philadelphia, 19104 [Recruiting]
  • Memphis, Tennessee, United States, St. Jude Children's Research Hospital, 38105 [Recruiting]
  • Nashville, Tennessee, United States, The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, 37203 [Recruiting]
  • San Antonio, Texas, United States, Methodist Children's Hospital/Texas Transplant Institute, 78229 [Recruiting]
  • Brussels, Belgium, Hopital Universitaire des Enfants Reine Fabiola (HUDERF) [Recruiting]
  • Toronto, Canada, The Hospital for Sick Children [Recruiting]
  • Regensburg, Germany, Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation [Recruiting]
  • Rome, Italy, Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS [Recruiting]
  • London, United Kingdom, Imperial College Healthcare NHS Trust, Hammersmith Hospital [Recruiting]

More info

View on ClinicalTrials.gov
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