A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Locations

5 United States sites

1 Italy site

1 Germany site

Age

> 18 Years

Study type

Observational

Gender

All

Interventions

Biological

Compensation

Unknown

About the study

This is a multi-site, observational study to evaluate the long-term safety and efficacy of
CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or Study CTX001-121
(NCT03745287).

participation requirements

– Subjects or legal representative or guardian (if applicable) must sign and date
informed consent form (ICF)

– Subjects must have received CTX001 infusion

participation restrictions

– There are no exclusion criteria

Locations

  • New York, New York, United States, Columbia University Medical Center (21+ years), 10032
  • New York, New York, United States, Columbia University Medical Center, 10032
  • Memphis, Tennessee, United States, St. Jude Children's Research Hospital, 38105
  • Nashville, Tennessee, United States, The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, 37203
  • San Antonio, Texas, United States, Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital, 78229
  • Regensburg, Germany, Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
  • Rome, Italy, Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS