A Long-term Follow-up Study in Participants Who Received CTX001
Public ClinicalTrials.gov record NCT04208529. 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 Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).
Study identification
- NCT ID
- NCT04208529
- Recruitment status
- Enrolling by invitation
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 160 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years and older
- 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
- Jan 20, 2021 (Actual)
- Primary completion
- Sep 30, 2039 (Estimated)
- Completion
- Sep 30, 2039 (Estimated)
- Last update posted
- Aug 11, 2026
2021 – 2039
United States locations
- U.S. sites
- 9
- U.S. states
- 7
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology | Chicago | Illinois | 60611 | — |
| Herbert Irving Pavilion - Hematology | New York | New York | 10032 | — |
| New York Presbyterian Hospital - Morgan Stanley Children's Hospital | New York | New York | 10032 | — |
| Levine Children's Hospital - Hematology | Charlotte | North Carolina | 28203 | — |
| The Children's Hospital of Philadelphia - Hematology | Philadelphia | Pennsylvania | 19104 | — |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | Tennessee | 37203 | — |
| Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital | 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 11 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04208529, 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 Aug 11, 2026 · Synced Oct 18, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04208529 live on ClinicalTrials.gov.