A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Public ClinicalTrials.gov record NCT03745287. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03745287
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Enrollment
- 63 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 12 Years to 35 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 27, 2018 (Actual)
- Primary completion
- Jul 7, 2025 (Actual)
- Completion
- Jul 7, 2025 (Actual)
- Last update posted
- Sep 16, 2026
2018 – 2025
United States locations
- U.S. sites
- 9
- U.S. states
- 6
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Lucile Packard Children's Hospital of Stanford University | Palo Alto | California | 94304 | — |
| Ann & Robert Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University of Illinois at Chicago Hospitals and Health Systems | Chicago | Illinois | 60612 | — |
| Columbia University Medical Center (21+ years) | New York | New York | 10032 | — |
| Columbia University Medical Center (≤21 years) | New York | New York | 10032 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
| The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers | Nashville | Tennessee | 37203 | — |
| Methodist Children's Hospital/Texas Transplant Institute | San Antonio | Texas | 78229 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 8 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03745287, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 16, 2026 · Synced Oct 23, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03745287 live on ClinicalTrials.gov.