Trial · NCT03745287
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
NCT03745287, Phase 2, Phase 3, Completed, Vertex Pharmaceuticals Incorporated
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease. Trials, record NCT03745287. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03745287
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
- NCT number
- NCT03745287
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 63 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Interventions
- Exa-cel
- First posted
- 2018-11-19
- Last updated
- 2026-09-16
Source ClinicalTrials.gov
Registered outcomes38
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Have Not Experienced Any Severe Vaso-occlusive Crisis (VOC) for at Least 12 Consecutive Months (VF12) After Exa-cel Infusion | From 60 days after last RBC transfusion up to 2 years after exa-cel infusion | A VOC is a condition of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. The percentage of participants who remain VOC free after achieving VF12 were data reported in the outcome measure. |
| Primary | Percentage of Participants Who Achieve Neutrophil Engraftment | Up to 24 months post exa-cel infusion. | Neutrophil engraftment is defined as the first day of 3 consecutive measurements of absolute neutrophil count (ANC)≥500/μL on 3 different days, within 42 days after exa-cel infusion without the use of unmodified CD34+ cells after reaching the nadir, defined as ANC \<500/µL. |
| Primary | Time to Neutrophil Engraftment for Participants Who Achieve Neutrophil Engraftment | Up to 24 months post exa-cel infusion. | Neutrophil engraftment is defined as the first day of 3 consecutive measurements of absolute neutrophil count (ANC)≥500/μL on 3 different days, without use of the unmodified CD34+ cells after reaching the nadir, defined as ANC\<500/μL. Time to neutrophil engraftment was calculated by the neutrophil engraftment date subtract exa-cel infusion date +1. |
| Primary | Time to Platelet Engraftment for Participants Who Achieve Platelet Engraftment | From Exa-cel infusion up to 2 years after exa-cel infusion | Platelet engraftment is defined as the first day of 3 consecutive measurements of unsupported (no platelet transfusions for the last 7 days) platelet ≥50,000/μL on 3 different days after Exa-cel infusion. For participants discharged early day 7 after the last platelet transfusion will be the day of platelet engraftment, as long as 3 subsequent and consecutive unsupported measurements on 3 different days are \>50,000/μL. For participants who have been discharged prior to platelet engraftment, it is recommended to collect blood every 2 to 3 days to obtain an accurate assessment of platelet engraftment. Time to platelet engraftment was defined as first of 3 consecutive measurements on 3 different days with platelet ≥50 × 109/L without a platelet transfusion for 7 consecutive days. |
| Primary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From receiving exa-cel infusion up to 2 years | |
| Primary | Transplant-related Mortality (TRM) Within 100 Days After Exa-cel Infusion | Within 100 days after exa-cel infusion | The transplant-related mortality is defined as death related to Busulfan and/or exa-cel infusion. The number and proportion of TRM participants who have died within 100 days, or with at least 100 days post exa-cel infusion. |
| Primary | Transplant-related Mortality Within 12 Months Post Exa-cel Infusion | Within 12 months post exa-cel infusion | The transplant-related mortality is defined as death related to Busulfan and/or exa-cel infusion. The number and proportion of TRM within 12 months will be summarized for participants who have died within 12 months, or with at least 12 months post exa-cel infusion. |
| Primary | All-cause Mortality | From exa-cel infusion up to 2 years | All-cause mortality from exa-cel infusion up to 2 years |
| Secondary | Percentage 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 2 years after exa-cel infusion | The HF12 means Free from inpatient hospitalization for severe vaso-occlusive crises (VOCs) and sustained for at least 12 months after exa-cel infusion. |
| Secondary | Relative Reduction From Baseline in Annualized Rate of Severe VOCs for Participants Who do Not Achieve VF12 up to 24 Months After Exa-cel Infusion | From 60 days after last RBC transfusion up to 2 years after exa-cel infusion | The VF12 defines absence of any severe vaso-occlusive crises (VOCs) for at least 12 consecutive months after exa-cel infusion. Only severe VOCs adjudicated by an outcome measure adjudication committee as meeting the protocol definition of severe VOCs were included in the analysis. Relative reduction from baseline was calculated as 100 % × (Baseline value - post-baseline value) / Baseline value. Annualized rate is calculated by the Total number of events/number of years. |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 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.
Papers9
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/NCT03745287
GET https://conductscience.org/api/trials/v1/NCT03745287/registered?page=2