NCT03745287CompletedPhase 2/3
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
A Phase 1/2/3 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
Source: ClinicalTrials.gov · Last updated 16 Sept 2026
Brief summary (registry, in English)
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants 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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2/3
- Study type
- Interventional
- Enrollment
- 63 (actual)
- First submitted
- 9 Nov 2018
- Start date
- 27 Nov 2018
- Primary completion date
- 7 Jul 2025
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Diseases
- Sickle cell disease
- NCT number
- NCT03745287
- EU CT number
- 2024-516067-83-00
- EudraCT number
- 2018-001320-19
What you commit to
- Study type: Interventional · Phase 2/3
- Participants enrolled: 63
- From 27 Nov 2018 to the primary completion date, 7 Jul 2025
- 16 sites in 7 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior hematopoietic stem cell transplant (HSCT)
Other illness
- Clinically significant and active bacterial, viral, fungal, or parasitic infection
- Other protocol defined inclusion/exclusion criteria may apply.
Lab values
- An available 10/10 human leukocyte antigen (HLA)-matched related donor
Who the study is looking for
- 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
Age in the registry: 12 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (16)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine Fabiola Coordinator of this site? Confirm its status | Brussels | Belgium | Completed |
| The Hospital for Sick Children Coordinator of this site? Confirm its status | Toronto | Canada | Unknown status |
| University Hospital Duesseldorf Coordinator of this site? Confirm its status | Düsseldorf | Germany | Unknown status |
| Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation Coordinator of this site? Confirm its status | Regensburg | Germany | Unknown status |
| Hopital Necker Enfants Malades Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Imperial College Healthcare Nhs Trust - Hammersmith Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Rome | Italy | Completed |
| University of Illinois at Chicago Hospitals and Health Systems Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| Ann & Robert Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
| The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Columbia University Medical Center (21+ years) Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Lucile Packard Children's Hospital of Stanford University Coordinator of this site? Confirm its status | Palo Alto | United States | Unknown status |
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Methodist Children's Hospital/Texas Transplant Institute Coordinator of this site? Confirm its status | San Antonio | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.