A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
Public ClinicalTrials.gov record NCT03655678. 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 of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
Study identification
- NCT ID
- NCT03655678
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Enrollment
- 59 participants
Conditions and interventions
Conditions
Interventions
- CTX001 Biological
Biological
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
- Sep 13, 2018
- Primary completion
- Nov 12, 2025
- Completion
- Nov 12, 2025
- Last update posted
- Dec 16, 2025
2018 – 2025
United States locations
- U.S. sites
- 6
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | — |
| Ann & Robert Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Columbia University Medical Center (21+ years) | New York | New York | 10032 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers | Nashville | Tennessee | 37203 | — |
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 NCT03655678, 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 Dec 16, 2025 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03655678 live on ClinicalTrials.gov.