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

Description

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.

Conditions

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

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Beta-Thalassemia
Intervention / Treatment

-

Contacts and Locations

New York

Columbia University Medical Center, New York, New York, United States, 10032

Charlotte

Atrium Health Levine Children's Hospital, Charlotte, North Carolina, United States, 28203

Nashville

SCRI at the Children's Hospital at TriStar Centennial, Nashville, Tennessee, United States, 37203

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Participants with TDT and SCD:
  • * Eligible for autologous stem cell transplant as per investigator's judgment.
  • * Participants with TDT:
  • * Diagnosis of TDT as defined by:
  • * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
  • * History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
  • * Participants with SCD:
  • * Diagnosis of severe SCD as defined by:
  • * Documented SCD genotypes
  • * History of at least two severe VOCs events per year for the previous two years prior to enrollment
  • * Participants with TDT and SCD:
  • * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
  • * Prior hematopoietic stem cell transplant (HSCT)
  • * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
  • * Participants with TDT:
  • * Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
  • * Participants with sickle cell β-thalassemia variant
  • * Participants with SCD:
  • * History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening

Ages Eligible for Study

12 Years to 35 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Vertex Pharmaceuticals Incorporated,

Study Record Dates

2025-02