Trial · NCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529, Phase 3, Enrolling by invitation, Vertex Pharmaceuticals Incorporated
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Long-term Follow-up Study in Participants Who Received CTX001. Trials, record NCT04208529. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04208529
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Long-term Follow-up Study in Participants Who Received CTX001
- NCT number
- NCT04208529
- Status
- Enrolling by invitation
- Phases
- Phase 3
- Enrollment
- 160 participants, estimated
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
- Interventions
- CTX001
- First posted
- 2019-12-23
- Last updated
- 2026-08-11
Source ClinicalTrials.gov
Registered outcomes41
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | New malignancies | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | New or worsening hematologic disorders | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | All-cause mortality | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | Serious adverse events (SAEs) | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | CTX001-related adverse events (AEs) | Signing of informed consent up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Total Hemoglobin (Hb) concentration over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Fetal Hemoglobin (HbF) concentration over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111 and 121 only | Up to 5 years post CTX001 infusion |
1 to 10 of 41Source ClinicalTrials.gov
See all 41 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04208529
GET https://conductscience.org/api/trials/v1/NCT04208529/registered?page=2