- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05951205
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype
February 28, 2024 updated by: Vertex Pharmaceuticals Incorporated
A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype
The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
Study Overview
Status
Not yet recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
12
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Medical Information
- Phone Number: 617-341-6777
- Email: medicalinfo@vrtx.com
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
Accepts Healthy Volunteers
No
Description
Key Inclusion Criteria:
- Participants with documented βS/βC (HbSC) genotype
- Participants must be eligible for autologous stem cell transplant as per investigator's judgment
Key Exclusion Criteria:
- A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
- Participants with prior hematopoietic stem cell transplant (HSCT)
- Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment.
Other protocol defined Inclusion/Exclusion criteria may apply.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Exa-cel
Participants will receive a single infusion of exa-cel (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene) through a central venous catheter.
|
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months
Time Frame: From 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion
|
From 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: From Signing of Informed Consent up to 24 Months After exa-cel Infusion
|
From Signing of Informed Consent up to 24 Months After exa-cel Infusion
|
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)
Time Frame: Within 42 Days After exa-cel Infusion
|
Within 42 Days After exa-cel Infusion
|
Time to Neutrophil Engraftment
Time Frame: Up to 24 months After exa-cel Infusion
|
Up to 24 months After exa-cel Infusion
|
Time to Platelet Engraftment
Time Frame: Up to 24 months After exa-cel Infusion
|
Up to 24 months After exa-cel Infusion
|
Incidence of Transplant-Related Mortality (TRM)
Time Frame: Up to 100 Days After exa-cel Infusion
|
Up to 100 Days After exa-cel Infusion
|
Incidence of Transplant-Related Mortality (TRM)
Time Frame: Within 12 Months After exa-cel Infusion
|
Within 12 Months After exa-cel Infusion
|
Incidence of All-cause Mortality
Time Frame: From Signing of Informed Consent up to 24 Months After exa-cel Infusion
|
From Signing of Informed Consent up to 24 Months After exa-cel Infusion
|
Proportion of Participants With No Severe Vaso-Occlusive Crises (VOCs) for At least 12 Months (VF12)
Time Frame: From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
Proportion of Participants Free from Inpatient Hospitalization For Severe VOCs Sustained for At least 12 Months (HF12)
Time Frame: From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
Relative Reduction in Annualized Rate of Severe VOCs
Time Frame: From Baseline up to 24 Months After exa-cel Infusion
|
From Baseline up to 24 Months After exa-cel Infusion
|
Duration of Severe VOC Free in Participants who Have Achieved VF12
Time Frame: From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
Relative Reduction in Rate of Inpatient Hospitalizations for Severe VOCs
Time Frame: From Baseline up to 24 Months After exa-cel Infusion
|
From Baseline up to 24 Months After exa-cel Infusion
|
Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs
Time Frame: From Baseline up to 24 Months After exa-cel Infusion
|
From Baseline up to 24 Months After exa-cel Infusion
|
Proportion of Participants With Sustained HbF >= 20 % for At least 3, 6, or 12 months
Time Frame: From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
|
Relative Reduction in Annualized Volume of RBC Transfusions
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
HbF Concentration Over Time
Time Frame: Up To 24 Months After exa-cel Infusion
|
Up To 24 Months After exa-cel Infusion
|
Total Hemoglobin (Hb) Concentration Over Time
Time Frame: Up To 24 Months After exa-cel Infusion
|
Up To 24 Months After exa-cel Infusion
|
Change In Reticulocyte Count Over Time
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Indirect Bilirubin Over Time
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Haptoglobin Over Time
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Lactate dehydrogenase (LDH) Over Time
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Time to First Detectable Haptoglobin
Time Frame: Up to 24 Months After exa-cel Infusion
|
Up to 24 Months After exa-cel Infusion
|
Time to First Normalized LDH
Time Frame: Up to 24 Months After exa-cel Infusion
|
Up to 24 Months After exa-cel Infusion
|
Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time
Time Frame: Up To 24 Months After exa-cel Infusion
|
Up To 24 Months After exa-cel Infusion
|
Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time
Time Frame: Up To 24 Months After exa-cel Infusion
|
Up To 24 Months After exa-cel Infusion
|
Change in Pain Scale (11-point numerical rating scale (NRS)) Assessment Over Time In Adults (>=18 Years)
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Over Time In Adults (>=18 Years)
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me)
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Pain Scale (11-point NRS) Assessment Over Time In Adolescents (12 to <18 years of age)
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in Pediatric Quality of Life Inventory (PedsQL; self-report and parent proxy versions) Generic Core In Adolescents (12 to <18 years of age)
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Change in PedsQL SCD module (self-report and parent proxy versions) In Adolescents (12 to <18 years of age)
Time Frame: From Baseline Up To 24 Months After exa-cel Infusion
|
From Baseline Up To 24 Months After exa-cel Infusion
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Estimated)
April 1, 2024
Primary Completion (Estimated)
December 1, 2029
Study Completion (Estimated)
December 1, 2029
Study Registration Dates
First Submitted
June 23, 2023
First Submitted That Met QC Criteria
July 14, 2023
First Posted (Actual)
July 18, 2023
Study Record Updates
Last Update Posted (Actual)
February 29, 2024
Last Update Submitted That Met QC Criteria
February 28, 2024
Last Verified
July 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX21-CTX001-171
- 2023-503247-34-00 (Other Identifier: EU CT)
- 2021-006375-41 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
IPD Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Sickle Cell Disease
-
Klein Buendel, Inc.National Institute on Minority Health and Health Disparities (NIMHD); Hilton...CompletedSickle Cell Disease | Sickle Cell Anemia in Children | Sickle Cell Thalassemia | Sickle Cell SC DiseaseUnited States
-
SangartCompletedSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | Hemoglobin SC Disease | Sickle Cell Disorders | Sickle Cell Hemoglobin C DiseaseUnited Kingdom, France, Jamaica, Lebanon
-
Nova Laboratories LimitedCompletedSickle Cell Disease | Sickle Cell Hemoglobin C | Sickle Cell-beta-thalassemia | Sickle-Cell; Hemoglobin Disease, ThalassemiaUnited Kingdom, Jamaica
-
SangartWithdrawnSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | Hemoglobin SC Disease | Sickle Cell Disorders | Sickle Cell Hemoglobin C DiseaseFrance, United Kingdom, Netherlands, Turkey, Bahrain, Belgium, Brazil, Lebanon, Qatar
-
University of British ColumbiaCompletedSickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS DiseaseCanada, Nepal
-
Sidney Kimmel Cancer Center at Thomas Jefferson...National Heart, Lung, and Blood Institute (NHLBI)TerminatedSickle Cell Anemia | Sickle Cell-hemoglobin C Disease | Sickle Cell-β0-thalassemiaUnited States
-
University of RegensburgRecruitingSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | HbS Disease | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SGermany, Austria
-
Centre Hospitalier Intercommunal CreteilRecruitingSickle-Cell Disease Nos With CrisisFrance
-
HemaQuest Pharmaceuticals Inc.TerminatedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Egypt, Canada, Jamaica
-
HemaQuest Pharmaceuticals Inc.CompletedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Canada, Egypt, Jamaica
Clinical Trials on Exa-cel
-
Vertex Pharmaceuticals IncorporatedCRISPR TherapeuticsEnrolling by invitationHematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-ThalassemiaUnited States, Canada, Italy, United Kingdom, Germany
-
Vertex Pharmaceuticals IncorporatedCRISPR TherapeuticsRecruitingHematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-ThalassemiaUnited States, Italy, Germany
-
Vertex Pharmaceuticals IncorporatedCRISPR TherapeuticsRecruitingHematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-ThalassemiaUnited States, Germany, Italy, United Kingdom, Canada
-
Vertex Pharmaceuticals IncorporatedCRISPR TherapeuticsActive, not recruitingHematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-ThalassemiaUnited States, United Kingdom, Germany, Canada, Italy
-
Vertex Pharmaceuticals IncorporatedCRISPR TherapeuticsRecruitingSickle Cell Disease | Hemoglobinopathies | Hematological Diseases | Hydroxyurea Failure | Hydroxyurea IntoleranceUnited States, Italy, United Kingdom, Germany
-
Vertex Pharmaceuticals IncorporatedCRISPR TherapeuticsActive, not recruitingSickle Cell Disease | Hemoglobinopathies | Hematological DiseasesUnited States, Belgium, Canada, France, Germany, Italy, United Kingdom
-
Nikkiso America, Inc.NAMSACompletedDialysis | Chronic Renal FailureUnited States
-
TruDiagnosticSRWRecruiting
-
Weill Medical College of Cornell UniversityAngiocrine BioscienceRecruiting