Education and Training

Radiation Therapy Residency

Medical Students

Medical Physics Residency

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

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.

Stanford is currently accepting patients for this trial.

Stanford Investigator(s):

Intervention(s):

  • biological: CTX001

Eligibility


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 the previous
   two years prior to enrollment

   - Eligible for autologous stem cell transplant as per investigators judgment

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

Ages Eligible for Study

12 Years - 35 Years

Genders Eligible for Study

All

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Recruiting