Casgevy (exagamglogene autotemcel) / Vertex, CRISPR Therap, Molecular Templates 
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10 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Casgevy (exagamglogene autotemcel) / Vertex, CRISPR Therap, Molecular Templates
2021-006390-37: A study to learn about the safety and efficacy of CTX001 (the "study drug product") to treat beta-thalassemia and severe sickle cell disease in patients

Not yet recruiting
3
18
Europe
Exagamglogene autotemcel (Exa-cel), CTX001, Dispersion for infusion
Vertex Pharmaceuticals Incorporated, VERTEX PHARMACEUTICALS INCORPORATED, Vertex Pharmaceuticals Incorporated,
Transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD), Transfusion-dependent β-thalassemia and severe sickle cell disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2021-002172-39: A study to learn about the safety and efficacy of CTX001 (the "study drug product") to treat beta-thalassemia in pediatric subjects Studio per comprendere la sicurezza e l'efficacia di CTX001 (il "prodotto in studio") nel trattamento della ß-talassemia in soggetti pediatrici

Not yet recruiting
3
12
Europe
filgrastim, CTX001, Plerixafor, Busulfan, [filgrastim], [CTX001], [Plerixafor], [Busulfan], Solution for injection, Dispersion for infusion, Concentrate for solution for injection/infusion
VERTEX PHARMACEUTICALS INCORPORATED, Vertex Pharmaceuticals Incorporated, Vertex Pharmaceuticals Incorporated
Transfusion-Dependent ß Thalassemia ß-talassemia trasfusione-dipendente, Transfusion-Dependent ß Thalassemia ß-talassemia trasfusione-dipendente, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2021-002173-26: A study to learn about the safety and efficacy of CTX001 (the "study drug product") to treat severe sickle cell disease in a pediatric subjects Uno studio per conoscere la sicurezza e l'efficacia di CTX001 (il "prodotto in studio") per il trattamento della malattia a cellule falciformi grave in soggetti pediatrici

Not yet recruiting
3
12
Europe
CTX001, Plerixafor, Busulfan, [CTX001], [Plerixafor], [Busulfan], Dispersion for infusion, Solution for injection, Concentrate for solution for injection/infusion
VERTEX PHARMACEUTICALS INCORPORATED, Vertex Pharmaceuticals Incorporated, Vertex Pharmaceuticals Incorporated
Severe Sickle Cell Disease (SCD) Malattia a cellule falciformi grave, Severe Sickle Cell Disease Malattia a cellule falciformi grave, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT05477563: Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Recruiting
3
26
Europe, US, RoW
CTX001, Exagamglogene autotemcel, Exa-cel
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics
Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Anemia, Sickle Cell Disease
02/25
02/25
NCT05329649: Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Recruiting
3
15
Europe, US
CTX001, Exagamglogene autotemcel, Exa-cel
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics
Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
05/26
05/26
NCT05356195: Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Recruiting
3
15
Europe, Canada, US
CTX001, Exagamglogene autotemcel, Exa-cel
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
05/26
05/26
NCT05951205: Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Not yet recruiting
3
12
NA
Exa-cel, Exagamglogene autotemcel, CTX001
Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
12/29
12/29
NCT04208529 / 2018-002935-88: A Long-term Follow-up Study in Participants Who Received CTX001

Enrolling by invitation
3
160
Europe, Canada, US
CTX001, Exagamglogene autotemcel, Exa-cel
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics
Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
09/39
09/39
CLIMB THAL-111, NCT03655678 / 2017-003351-38: A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

Active, not recruiting
2/3
59
Europe, Canada, US
CTX001, Exagamglogene autotemcel, Exa-cel
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
12/25
12/25
CLIMB SCD-121, NCT03745287 / 2018-001320-19: A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Active, not recruiting
2/3
63
Europe, Canada, US
CTX001, Exagamglogene autotemcel, Exa-cel
Vertex Pharmaceuticals Incorporated, CRISPR Therapeutics
Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
07/25
07/25

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