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CTX001

BIOLOGICAL6 trials

Sponsors

Vertex Pharmaceuticals Incorporated, Complement Therapeutics

Conditions

Beta-ThalassemiaGenetic Diseases, InbornGeographic Atrophy Secondary to Age-related Macular DegenerationHematologic DiseasesHematological DiseasesHemoglobinopathiesHydroxyurea FailureHydroxyurea Intolerance

Phase 1

An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
RecruitingNCT07392255
Complement TherapeuticsGeographic Atrophy Secondary to Age-related Macular Degeneration
Start: 2025-12-30End: 2032-06-30Target: 75Updated: 2026-06-22

Phase 2

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
CompletedNCT03655678
Vertex Pharmaceuticals IncorporatedBeta-Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases +2
Start: 2018-09-14End: 2025-11-13Updated: 2025-12-17

Phase 3

A Long-term Follow-up Study in Participants Who Received CTX001
Enrolling by invitationNCT04208529
Vertex Pharmaceuticals IncorporatedBeta-Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases +4
Start: 2021-01-20End: 2039-09-30Target: 160Updated: 2026-08-11
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Active, not recruitingNCT05329649
Vertex Pharmaceuticals IncorporatedHematological Diseases, Hemoglobinopathies, Hydroxyurea Failure +2
Start: 2022-05-02End: 2027-06-06Updated: 2026-07-01
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Active, not recruitingNCT05356195
Vertex Pharmaceuticals IncorporatedBeta-Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases +2
Start: 2022-05-03End: 2027-11-14Updated: 2026-07-01
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
RecruitingNCT05477563
Vertex Pharmaceuticals IncorporatedBeta-Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases +4
Start: 2022-08-02End: 2027-06-09Target: 26Updated: 2026-03-23

Related Papers

Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.
2026-06-11
Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-Thalassemia.
2026-06-07
Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel.
2025-12-013 citations
Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.
2025-12-012 citations
Rheological Comparison of Red Cells from Individuals with SCD Treated with Gene Therapy, Haploidentical Transplantation or Hydroxyurea
Blood2024-11-052 citations
Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
New England Journal of Medicine2024-04-24136 citations
Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches
International Journal of Molecular Sciences2024-02-2040 citations
5612617 EFFICACY AND SAFETY OF A SINGLE DOSE OF EXAGAMGLOGENE AUTOTEMCEL FOR TRANSFUSION-DEPENDENT-THALASSEMIA AND SEVERE SICKLE CELL DISEASE
HemaSphere2023-04-013 citations
Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
Blood2022-11-1510 citations
Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin)
Therapeutic Advances in Hematology2022-01-0127 citations
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
New England Journal of Medicine2020-12-051721 citations
Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells
Blood2020-11-0449 citations