A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529Phase 3Enrolling by invitation
- Lead sponsor
- Vertex Pharmaceuticals Incorporated (Industry)
- Enrollment
- 160 (estimated)
- Start date
- 20 Jan 2021
- Primary completion
- 30 Sep 2039 (estimated)
- Study completion
- 30 Sep 2039
- Last update posted
- 11 Aug 2026
Design
- Study type
- Interventional
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None
- Arms
- 1
Interventions
- CTX001 Biological
Conditions and primary outcome
- Beta-Thalassemia
- Thalassemia
- Sickle Cell Disease
- Hematologic Diseases
- Hemoglobinopathies
- Genetic Diseases, Inborn
- Sickle Cell Anemia
Primary outcome
New malignancies (Signing of informed consent up to 15 years post CTX001 infusion); New or worsening hematologic disorders (Signing of informed consent up to 15 years post CTX001 infusion); All-cause mortality (Signing of informed consent up to 15 years post CTX001 infusion); Serious adverse events (SAEs) (Signing of informed consent up to 15 years post CTX001 infusion); CTX001-related adverse events (AEs) (Signing of informed consent up to 15 years post CTX001 infusion)
Posted results on ClinicalTrials.gov
Results posted on ClinicalTrials.gov: No
Results from company and journal sources
No editor-confirmed results are on record for this study.
Publications
3 on PubMedCorrection of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-Thalassemia.
American journal of hematology · 2026 Aug
PubMed 42252696 (opens in a new tab)DOI 10.1002/ajh.70382 (opens in a new tab)
Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel.
Blood advances · 2025 Dec 23
PubMed 40862696 (opens in a new tab)DOI 10.1182/bloodadvances.2025016702 (opens in a new tab)
Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.
Blood advances · 2025 Dec 23
PubMed 40857358 (opens in a new tab)DOI 10.1182/bloodadvances.2025016701 (opens in a new tab)
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.
CT-VCA trial quality
About CT-VCALoading CT-VCA scores…
Data integrity
Compare what the registry, company releases, FDA documents and publications say about this study.
Drugs on HealthTrade
Source
- NCT04208529 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.
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