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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Vertex Pharmaceuticals Incorporated
Study ID
NCT03745287
Phase
PHASE2/PHASE3
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 35 Years
Healthy Volunteers
Not accepted

Interventions

  • CTX001 — BIOLOGICAL
    Administered by IV infusion following myeloablative conditioning with busulfan.

Study Details

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.

Key Dates

Start date
Nov 27, 2018
Status verified
Aug 2025
Primary completion
Jul 7, 2025
Completion
Jul 7, 2025

Study Design

Enrollment
63 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CTX001
    CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Subjects will receive a single infusion of CTX001 through a central venous catheter.

Primary Outcome Measure

Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12) [ Time Frame: From 60 days after last RBC transfusion up to 2 years after CTX001 infusion ]

Locations (9)

FacilityCityStateZIPSite coordinators
Lucile Packard Children's Hospital of Stanford UniversityPalo AltoCalifornia94304-
Ann & Robert Lurie Children's Hospital of ChicagoChicagoIllinois60611-
University of Illinois at Chicago Hospitals and Health SystemsChicagoIllinois60612-
Columbia University Medical Center (≤21 years)New YorkNew York10032-
Columbia University Medical Center (21+ years)New YorkNew York10032-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
St. Jude Children's Research HospitalMemphisTennessee38105-
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood CancersNashvilleTennessee37203-
Methodist Children's Hospital/Texas Transplant InstituteSan AntonioTexas78229-

Find similar trials in Palo Alto, CA

By condition
Sickle cell disease
By research site
Lucile Packard Children's Hospital of Stanford University· Palo Alto, CAAnn & Robert Lurie Children's Hospital of Chicago· Chicago, ILUniversity of Illinois at Chicago Hospitals and Health Systems· Chicago, ILColumbia University Medical Center (≤21 years)· New York, NYColumbia University Medical Center (21+ years)· New York, NYChildren's Hospital of Philadelphia· Philadelphia, PA

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