October, 10 2019

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


7 United States sites, 1 Belgium site, 1 Canada site, 1 Germany site, 1 Italy site


18 to 35 Years




Phase 1/Phase 2

About the study

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12
subjects 18 to 35 years of age 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.

Study Type



Phase 1/Phase 2


18 to 35 Years





Inclusion Criteria

– Subjects ≥ 18 and ≤ 35 years of age

– Diagnosis of severe sickle cell disease as defined by:

– Documented βS/βS 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


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


  • Biological

Outcome measures

  • All-cause mortality
  • Frequency and severity of collected adverse events (AEs)
  • Incidence of transplant-related mortality (TRM) within 100 days after CTX001 infusion
  • Incidence of TRM within 1 year after CTX001 infusion
  • Proportion of subjects with engraftment (absolute neutrophil count [ANC] ≥500/µL for three consecutive days)
  • Proportion of subjects with HbF ≥20%, sustained for at least 3 months at the time of analysis, starting 6 months after CTX001 infusion
  • Time to engraftment


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

Contact Research Team

  • Medical Information

More info

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