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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-512654-19-00 | Other Identifier | EU CT Number |
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| Name | Class |
|---|---|
| CRISPR Therapeutics | INDUSTRY |
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This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia [TDT] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease [SCD] studies).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CTX001 | Experimental | All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151 and CTX001-161) after CTX001 infusion will be asked to participate in this long-term follow-up study. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTX001 | Biological | CTX001 infusion. |
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| Measure | Description | Time Frame |
|---|---|---|
| New malignancies | Signing of informed consent up to 15 years post CTX001 infusion | |
| New or worsening hematologic disorders | Signing of informed consent up to 15 years post CTX001 infusion | |
| All-cause mortality | Signing of informed consent up to 15 years post CTX001 infusion | |
| Serious adverse events (SAEs) | Signing of informed consent up to 15 years post CTX001 infusion | |
| CTX001-related adverse events (AEs) | Signing of informed consent up to 15 years post CTX001 infusion |
| Measure | Description | Time Frame |
|---|---|---|
| TDT and SCD: Total Hemoglobin (Hb) concentration over time | Up to 15 years post CTX001 infusion | |
| TDT and SCD: Fetal Hemoglobin (HbF) concentration over time | Up to 15 years post CTX001 infusion | |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | United States | ||
| Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42252696 | Derived | Sheth S, Corbacioglu S, de la Fuente J, Algeri M, Rupprecht J, Kuo KHM, Shah AJ, Lang P, Merkeley H, Carpenter B, Mapara MY, Liem RI, Grupp S, Chopra Y, Li AM, Kwiatkowski JL, Kirby-Allen M, Cappellini MD, Kattamis A, Zairis S, Liu T, Hobbs W, Frangoul H, Locatelli F, Meisel R; CLIMB THAL-111 and CLIMB-131 Study Groups. Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent beta-Thalassemia. Am J Hematol. 2026 Aug;101(8):1969-1979. doi: 10.1002/ajh.70382. Epub 2026 Jun 7. | |
| 40862696 |
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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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Long Term Safety Follow-up
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| TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time |
| Up to 15 years post CTX001 infusion |
| TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time | Up to 15 years post CTX001 infusion |
| TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111 and 121 only | Up to 5 years post CTX001 infusion |
| TDT and SCD: Change in PROs over time in participants ≥18 years of age assessed using functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) questionnaire for participants from study 111, 121 and 161 only | Up to 5 years post CTX001 infusion |
| TDT and SCD: Change in PROs over time in participants <18 years assessed using EQ-5D-Youth (EQ-5D-Y) from study 111,121,141 and 151 only | Up to 5 years post CTX001 infusion |
| TDT and SCD: Change in PROs over time in participants <18 years assessed using pediatric quality of life inventory (PedsQL) Core | Up to 5 years post CTX001 infusion |
| TDT: Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12) | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| TDT: Proportion of participants achieving transfusion independence for at least 6 consecutive months (TI6) | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| TDT: Proportion of participants achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized volume of RBC transfusions starting after month 10 after CTX001 infusion for participants who have not achieved TI12 | From Month 10 up to 15 years post-CTX001 infusion |
| TDT: Duration of transfusion free in participants who have achieved TI12 | From 60 days after last RBC transfusion up to 15 years post CTX001 infusion |
| TDT: Relative reduction from baseline in annualized volume of RBC transfusions starting after Month 10 after CTX001 infusion for participants who have not achieved TI12 | From Month 10 up to 15 years post-CTX001 infusion |
| TDT: Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin for beta-Thalassemia participants | Up to 8 years post CTX001 infusion for LIC; up to 5 years post CTX001 infusion for CIC and up to 15 years post CTX001 infusion for ferritin |
| TDT: Proportion of participants receiving iron removal therapy over time | Up to 15 years post CTX001 infusion |
| SCD: Proportion of participants who have not experienced any severe vaso-occlusive crises (VOC) for at least 12 consecutive months (VF12) | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Proportion of participants with SCD free from inpatient hospitalization for severe VOCs sustained for at least 12 months (HF12) | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Proportion of participants with at least 90 percent (%), 80%, 75% or 50% reduction in annualized rate of severe VOCs | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Relative change from baseline in annualized rate of severe VOCs | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Duration of severe VOC free in participants who have achieved VF12 | From 60 days after last RBC transfusion up to 15 years post CTX001 infusion |
| SCD: Relative change from baseline in rate of inpatient hospitalizations for severe VOCs | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Relative change from baseline in annualized duration of hospitalization for severe VOCs | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Proportion of participants with sustained HbF ≥20% for at least 3 months | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Proportion of participants with sustained HbF ≥20% for at least 6 months | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Proportion of participants with sustained HbF ≥20% for at least 12 months | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion |
| SCD: Change in volume of RBCs transfused for SCD-related indications over time | Up to 15 years post CTX001 infusion |
| SCD: Change from baseline in reticulocytes/erythrocytes over time | From baseline up to 15 years post CTX001 infusion |
| SCD: Change from baseline in lactate dehydrogenase (LDH) over time | From baseline up to 15 years post CTX001 infusion |
| SCD: Change from baseline in haptoglobin over time | From baseline up to 15 years post CTX001 infusion |
| SCD: Change from baseline in total bilirubin over time | From baseline up to 15 years post CTX001 infusion |
| SCD: Change from baseline in indirect bilirubin over time | From baseline up to 15 years post CTX001 infusion |
| SCD: Change in SCD-specific PROs over time in participants ≥18 years of age assessed using adult sickle cell quality of life measurement system (ASCQ-Me) (participants from Study 121 and 161 only) | Up to 5 years post CTX001 infusion |
| SCD: Change in SCD-specific PROs over time in participants <18 years of age assessed using PedsQL Generic Core SCD module from studies 111,121,141,151 and 161 | Up to 5 years post CTX001 infusion |
| SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS) | Up to 5 years post CTX001 infusion |
| SCD: Change in PROs over time assessed using Wong Baker FACES pain scale | Up to 5 years post CTX001 infusion |
| SCD: Change in PROs over time using face, legs, activity, cry, consolability (FLACC) behavioral pain scale | Up to 5 years post CTX001 infusion |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Herbert Irving Pavilion - Hematology | New York | New York | 10032 | United States |
| New York Presbyterian Hospital - Morgan Stanley Children's Hospital | New York | New York | 10032 | United States |
| Levine Children's Hospital - Hematology | Charlotte | North Carolina | 28203 | United States |
| The Children's Hospital of Philadelphia - Hematology | 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 |
| Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital | San Antonio | Texas | 78229 | United States |
| Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology | Brussels | Belgium |
| Hospital for Sick Children - Hematology | Toronto | Canada |
| Toronto General Hospital - Hematology | Toronto | Canada |
| St. Paul's Hospital - Hematology | Vancouver | Canada |
| University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology | Düsseldorf | Germany |
| Center for Pediatric Clinical Studies (CPCS) | Klinik Für Kinder- Und Jugendmedizin | Germany |
| Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine | Regensburg | Germany |
| IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica | Rome | Italy |
| Great Ormond Street Hospital for Children | London | United Kingdom |
| Hammersmith Hospital - Haematology Dept | London | United Kingdom |
| University College London Hospital NHS Foundation - Main | London | United Kingdom |
| Derived |
| Fuente J, Frangoul H, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara MY, Liem RI, Rupprecht J, Kuo KHM, Merkeley H, Algeri M, Smith W, Kohli P, Li N, Rubin J, Zhang S, Hobbs W, Locatelli F. Improvements in health-related quality of life in patients with transfusion-dependent beta-thalassemia after exagamglogene autotemcel. Blood Adv. 2025 Dec 23;9(24):6502-6510. doi: 10.1182/bloodadvances.2025016702. |
| 40857358 | Derived | Sharma A, Locatelli F, Bhatia M, Molinari L, Mapara MY, Liem RI, Dedeken L, Wall D, Eckrich MJ, Kuo KHM, Smith W, Imren S, Kohli P, Li N, Liu T, Rubin J, Hobbs W, Grupp SA, Frangoul H. Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel. Blood Adv. 2025 Dec 23;9(24):6481-6490. doi: 10.1182/bloodadvances.2025016701. |
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| D013789 | Thalassemia |
| D000755 | Anemia, Sickle Cell |
| D006402 | Hematologic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006425 | Hemic and Lymphatic Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000729927 | exagamglogene autotemcel |
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