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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-002173-26 | EudraCT Number |
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| Name | Class |
|---|---|
| CRISPR Therapeutics | INDUSTRY |
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This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CTX001 | Experimental | CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTX001 | Biological | Administered by intravenous infusion following myeloablative conditioning with busulfan. |
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| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12) | Up to 24 Months After CTX001 Infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants Free from Inpatient Hospitalization for Severe VOCs for at Least 12 Months (HF12) | Up to 24 Months After CTX001 Infusion | |
| Relative Reduction in Annualized Rate of Severe VOCs | From Baseline up to 24 Months After CTX001 Infusion |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Levine Children's Hospital - Hematology | Charlotte | North Carolina | 28203 | United States | ||
| The Children's Hospital of Philadelphia - Hematology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42274009 | Derived | Frangoul H, de la Fuente J, Chopra Y, Meisel R, Amrolia PJ, Algeri M, Sharma A, Cappellini MD, Corbacioglu S, Kattamis A, Lobitz S, de Montalembert M, Rondelli D, Sheth S, Steinberg MH, Walters MC, Boerner K, Liu T, Zairis S, Hobbs W, Grupp SA, Locatelli F; CLIMB THAL-141 and CLIMB SCD-151 Study Groups. Exa-cel in Children with Transfusion-Dependent beta-Thalassemia or Sickle Cell Disease. N Engl J Med. 2026 Jun 11. doi: 10.1056/NEJMoa2603387. Online ahead of print. |
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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
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| Duration of Severe VOC Free in Participants who Have Achieved VF12 | Up to 24 Months After CTX001 Infusion |
| Relative Reduction in Annualized Rate of Inpatient Hospitalizations for Severe VOCs | From Baseline up to 24 Months After CTX001 Infusion |
| Proportion of Participants With Sustained Fetal Hemoglobin (HbF) ≥20 Percent (%) for at Least 3 Months | Up to 24 Months After CTX001 Infusion |
| Proportion of Participants With Sustained HbF ≥20% for at Least 6 Months | Up to 24 Months After CTX001 Infusion |
| Proportion of Participants With Sustained HbF ≥20% for at Least 12 Months | Up to 24 Months After CTX001 Infusion |
| Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time | Up to 24 Months After CTX001 Infusion |
| Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time | Up to 24 Months After CTX001 Infusion |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Signing of Informed Consent up to 24 Months After CTX001 Infusion |
| 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 |
| Time to Engraftment | Up to 24 Months After CTX001 Infusion |
| Incidence of Transplant-related Mortality (TRM) Within 100 Days After CTX001 Infusion | Within 100 Days After CTX001 infusion |
| Incidence of TRM Within 12 Months After CTX001 Infusion | Within 12 Months After Infusion |
| Incidence of All-cause Mortality | From Signing of Informed Consent up to 24 Months After CTX001 Infusion |
| Relative Reduction in Annualized Duration of Hospitalization for Severe VOCs | From Baseline up to 24 Months After CTX001 Infusion |
| Proportion of Participants With Sustained HbF ≥30% for at Least 3 Months | Up to 24 Months After CTX001 Infusion |
| Proportion of Participants With Sustained HbF ≥30% for at Least 6 Months | Up to 24 Months After CTX001 Infusion |
| Proportion of Participants With Sustained HbF ≥30% for at Least 12 Months | Up to 24 Months After CTX001 Infusion |
| Time for Participants to Reach HbF ≥20% | Up to 24 Months After CTX001 Infusion |
| Time for Participants to Reach HbF ≥30% | Up to 24 Months After CTX001 Infusion |
| Relative Reduction from Baseline in Annualized Volume and Episodes of RBC Transfusions for SCD-related indications starting after Month 12 post-CTX001 infusion | Up to 24 Months After CTX001 Infusion |
| HbF Concentrations Over Time | Up to 24 Months After CTX001 Infusion |
| Hemoglobin (Hb) Concentrations Over Time | Up to 24 Months After CTX001 Infusion |
| Change in Reticulocyte Count Over Time | From Baseline up to 24 Months After CTX001 Infusion |
| Change in Indirect Bilirubin Over Time | From Baseline up to 24 Months After CTX001 Infusion |
| Change in Haptoglobin Over Time | From Baseline up to 24 Months After CTX001 Infusion |
| Proportion of Participants with Detectable Haptoglobin Over Time | Up to 24 Months After CTX001 Infusion |
| Change in Lactate Dehydrogenase (LDH) Over Time | From Baseline (Pre-infusion) up to 24 Months After CTX001 Infusion |
| Proportion of Participants with Normalized LDH Over Time | Up to 24 Months After CTX001 Infusion |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | Tennessee | 37203 | United States |
| University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology | Düsseldorf | Germany |
| IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica | Rome | Italy |
| St.Mary's Hospital - Haematology Dept | London | United Kingdom |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D006453 | Hemoglobinopathies |
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000729927 | exagamglogene autotemcel |
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