Trial · NCT05477563
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
NCT05477563, Phase 3, Recruiting, Vertex Pharmaceuticals Incorporated
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease. Trials, record NCT05477563. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05477563
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
- NCT number
- NCT05477563
- Status
- Recruiting
- Phases
- Phase 3
- Enrollment
- 26 participants, estimated
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
- Interventions
- CTX001
- First posted
- 2022-07-28
- Last updated
- 2026-03-23
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Fetal Hemoglobin (HbF) Concentration Over Time | Up to 12 Months After CTX001 Infusion | |
| Primary | Total Hemoglobin (Hb) Concentration Over Time | Up to 12 Months After CTX001 Infusion | |
| Secondary | TDT and SCD: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Signing of Informed Consent up to 12 Months After CTX001 Infusion | |
| Secondary | TDT and SCD: 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 | TDT and SCD: Time to Engraftment | Up to 12 Months After CTX001 Infusion | |
| Secondary | TDT and SCD: Incidence of Transplant-Related Mortality (TRM) Within 100 Days After CTX001 Infusion | Within 100 Days After CTX001 Infusion | |
| Secondary | TDT and SCD: Incidence of TRM Within 12 Months After CTX001 Infusion | Within 12 Months After CTX001 Infusion | |
| Secondary | TDT and SCD: Incidence of All-cause Mortality | From Signing of Informed Consent up to 12 Months After CTX001 Infusion | |
| Secondary | TDT and SCD: Relative Reduction in Annualized Volume of RBC Transfusions | From Day 60 up to 12 Months After CTX001 Infusion | |
| Secondary | TDT and SCD: Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time | Up to 12 Months After CTX001 Infusion |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 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.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT05477563
GET https://conductscience.org/api/trials/v1/NCT05477563/registered