Trial · NCT05356195
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
NCT05356195, Phase 3, Active not recruiting, Vertex Pharmaceuticals Incorporated
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT). Trials, record NCT05356195. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05356195
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
- NCT number
- NCT05356195
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 16 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- Interventions
- CTX001
- First posted
- 2022-05-02
- Last updated
- 2026-07-01
- Other registries' IDs
- eudract:2021-002172-39
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12) | Up to 24 Months After CTX001 Infusion | |
| Secondary | Proportion of Participants Achieving at Least 95 Percent (%), 90%, 85%, 75% and 50% Reduction in Annualized Transfusions | From Baseline up to 24 Months After CTX001 Infusion | |
| Secondary | Transfusion Free Duration for Participants who Achieve TI12 | Up to 24 Months After CTX001 Infusion | |
| Secondary | Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time | Up to 24 Months After CTX001 Infusion | |
| Secondary | Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time | Up to 24 Months After CTX001 Infusion | |
| Secondary | Change in Fetal Hemoglobin Concentration Over Time | From Baseline (Pre-transfusion) up to 24 Months After CTX001 Infusion | |
| Secondary | Change in Total Hemoglobin Concentration Over Time | From Baseline (Pre-transfusion) up to 24 Months After CTX001 Infusion | |
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Signing of Informed Consent up to 24 Months After CTX001 Infusion | |
| Secondary | Proportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count [ANC] ≥500 per Microliter [mcgL] on 3 Different Days) | Within 42 Days After CTX001 Infusion | |
| Secondary | Time to Engraftment | Up to 24 Months After CTX001 Infusion |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 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.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 42274009 | DERIVED |
Source PubMed
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/NCT05356195
GET https://conductscience.org/api/trials/v1/NCT05356195/registered