Clinical Trials Logo
  1. Home
  2. Hemoglobinopathies
  3. NCT05356195

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Hematologic Diseases Clinical Trial

Official title:
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia

Clinical Trial Summary

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05356195
Study type Interventional
Source Vertex Pharmaceuticals Incorporated
Contact Medical Information
Phone 617-341-6777
Email medicalinfo@vrtx.com
Status Recruiting
Phase Phase 3
Start date May 3, 2022
Completion date May 2026
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT04094844 - Health Information Technology System ("Roadmap 2.0") in the Context of Hematopoietic Cell Transplantation N/A
Completed NCT04474678 - Quality Improvement Project - "My Logbook! - I Know my Way Around!"; ("Mein Logbuch - Ich Kenne Mich Aus!") N/A
Recruiting NCT03918343 - Lipopolysaccharide Metabolism and Identification of Potential Biomarkers Predictive of Graft-versus-host Disease After Allogeneic Stem Cell Transplantation N/A
Completed NCT01445561 - Ultra Low Dose Interleukin-2 in Healthy Volunteers Phase 1
Recruiting NCT06148610 - Evaluation of the Impact of the Use of NewSpringForMe on Transplanted Patients' Quality of Life and Support
Completed NCT04168983 - Impact of Sophrology on the Pain Felt During a Bone Marrow Aspiration and Biopsy N/A
Not yet recruiting NCT05969821 - Clonal Hematopoiesis of Immunological Significance
Withdrawn NCT04282174 - CD34+ Enriched Transplants From HLA-Compatible Patients With Hematologic Malignancies Phase 2
Completed NCT01108159 - Integrated Whole-Genome Analysis of Hematologic Disorders
Completed NCT00800839 - Busulfan and Fludarabine Followed by Post-transplant Cyclophosphamide Phase 2
Completed NCT00213239 - A Dose Finding Study of Remifentanil and Propofol for Lumbar Punctures in Children Phase 1/Phase 2
Terminated NCT00208949 - A Comparison of Dendritic Cell Content and T-Cell Phenotype Between Granulocyte Colony-Stimulating Factor (G-CSF) or G-CSF + Granulocyte Macrophage (GM)-CSF Phase 2
Terminated NCT00176826 - T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders Phase 2/Phase 3
Completed NCT00208962 - Allogeneic Cell Therapy for Adults With Hematologic Malignancies Phase 2
Completed NCT00000603 - Cord Blood Stem Cell Transplantation Study (COBLT) Phase 2
Completed NCT00000587 - Erythropoietin for Anemia Due to Zidovudine in Human Immunodeficiency Virus Infection Phase 2
Active, not recruiting NCT03655678 - A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia Phase 2/Phase 3
Recruiting NCT05487794 - Effect of Dose Fractionation of Testosterone Cypionate on Transgender Men With Erythrocytosis N/A
Completed NCT03611257 - Effect of dRAST on Treatment for Bacteremia in Patients With Hematologic Diseases N/A
Completed NCT02827149 - High Resolution Donor Recipient HLA Matching Level in Unrelated HSCT