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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-503247-34-00 | Other Identifier | EU CT number | |
| 2021-006375-41 | EudraCT Number |
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Modification to the clinical development plan
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Exa-cel | Experimental | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Exa-cel | Biological | Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
| 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) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C000729927 | exagamglogene autotemcel |
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| Within 42 Days After exa-cel Infusion |
| Time to Neutrophil Engraftment | Up to 24 months After exa-cel Infusion |
| Time to Platelet Engraftment | Up to 24 months After exa-cel Infusion |
| Incidence of Transplant-Related Mortality (TRM) | Up to 100 Days After exa-cel Infusion |
| Incidence of Transplant-Related Mortality (TRM) | Within 12 Months After exa-cel Infusion |
| Incidence of All-cause Mortality | 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) | 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) | From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion |
| Relative Reduction in Annualized Rate of Severe VOCs | From Baseline up to 24 Months After exa-cel Infusion |
| Duration of Severe VOC Free in Participants who Have Achieved VF12 | 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 | From Baseline up to 24 Months After exa-cel Infusion |
| Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs | 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 | From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion |
| Relative Reduction in Annualized Volume of RBC Transfusions | From Baseline Up To 24 Months After exa-cel Infusion |
| HbF Concentration Over Time | Up To 24 Months After exa-cel Infusion |
| Total Hemoglobin (Hb) Concentration Over Time | Up To 24 Months After exa-cel Infusion |
| Change In Reticulocyte Count Over Time | From Baseline Up To 24 Months After exa-cel Infusion |
| Change in Indirect Bilirubin Over Time | From Baseline Up To 24 Months After exa-cel Infusion |
| Change in Haptoglobin Over Time | From Baseline Up To 24 Months After exa-cel Infusion |
| Change in Lactate dehydrogenase (LDH) Over Time | From Baseline Up To 24 Months After exa-cel Infusion |
| Time to First Detectable Haptoglobin | Up to 24 Months After exa-cel Infusion |
| Time to First Normalized LDH | Up to 24 Months After exa-cel Infusion |
| Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time | 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 | 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) | 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) | From Baseline Up To 24 Months After exa-cel Infusion |
| Change in Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) | 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) | 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) | 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) | From Baseline Up To 24 Months After exa-cel Infusion |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |