Trial · NCT05951205
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
NCT05951205, Phase 3, Withdrawn, Vertex Pharmaceuticals Incorporated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 Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype. Trials, record NCT05951205. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05951205
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 Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
- NCT number
- NCT05951205
- Status
- Withdrawn
- Phases
- Phase 3
- Enrollment
- 0 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Sickle Cell Disease
- Interventions
- Exa-cel
- First posted
- 2023-07-18
- Last updated
- 2026-03-25
- Other registries' IDs
- eudract:2021-006375-41
Source ClinicalTrials.gov
Registered outcomes32
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months | From 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion | |
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Signing of Informed Consent up to 24 Months After exa-cel Infusion | |
| Secondary | Proportion of Participants With Neutrophil Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days) | Within 42 Days After exa-cel Infusion | |
| Secondary | Time to Neutrophil Engraftment | Up to 24 months After exa-cel Infusion | |
| Secondary | Time to Platelet Engraftment | Up to 24 months After exa-cel Infusion | |
| Secondary | Incidence of Transplant-Related Mortality (TRM) | Up to 100 Days After exa-cel Infusion | |
| Secondary | Incidence of Transplant-Related Mortality (TRM) | Within 12 Months After exa-cel Infusion | |
| Secondary | Incidence of All-cause Mortality | From Signing of Informed Consent up to 24 Months After exa-cel Infusion | |
| Secondary | Proportion of Participants With No Severe Vaso-Occlusive Crises (VOCs) for At least 12 Months (VF12) | From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion | |
| Secondary | Proportion of Participants Free from Inpatient Hospitalization For Severe VOCs Sustained for At least 12 Months (HF12) | From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 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/NCT05951205
GET https://conductscience.org/api/trials/v1/NCT05951205/registered?page=2