Trials / Enrolling By Invitation
Enrolling By InvitationNCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001
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)
- Status
- Enrolling By Invitation
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 160 (estimated)
- Sponsor
- Vertex Pharmaceuticals Incorporated · Industry
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- Not accepted
Summary
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).
Conditions
- Beta-Thalassemia
- Thalassemia
- Sickle Cell Disease
- Hematologic Diseases
- Hemoglobinopathies
- Genetic Diseases, Inborn
- Sickle Cell Anemia
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | CTX001 | CTX001 infusion. |
Timeline
- Start date
- 2021-01-20
- Primary completion
- 2039-09-30
- Completion
- 2039-09-30
- First posted
- 2019-12-23
- Last updated
- 2026-03-25
Locations
20 sites across 6 countries: United States, Belgium, Canada, Germany, Italy, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04208529. Inclusion in this directory is not an endorsement.