RecruitingPhase 3NCT05477563

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

Sponsor

Vertex Pharmaceuticals Incorporated

Enrollment

26 participants

Start Date

Aug 2, 2022

Study Type

INTERVENTIONAL

Conditions

Beta-Thalassemia Sickle Cell Disease Hematologic Diseases Thalassemia Hemoglobinopathies Genetic Diseases, Inborn Sickle Cell Anemia

Summary

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Eligibility

Min Age: 12 YearsMax Age: 35 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called CTX001 for people with beta-thalassemia, genetic diseases, inborn, and other related conditions. The study is currently recruiting participants at 6 locations. People eligible for this study include aged 12 Years to 35 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALCTX001

Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan

Locations(6)

New York Presbyterian Hospital - Morgan Stanley Children's Hospital

New York, New York, United States

Levine Children's Hospital - Hematology

Charlotte, North Carolina, United States

TriStar Medical Group Children's Specialists - Pediatric Oncology

Nashville, Tennessee, United States

University Hospital Dusseldorf - 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

King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology

Al Mathar Ash Shamali, Saudi Arabia

View Full Details on ClinicalTrials.gov

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NCT05477563

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