RECRUITING

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

Conditions

Beta-Thalassemia Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

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

Quick Facts

Study Start:2022-05-03

Study Completion:2026-05

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05356195

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.

Ages Eligible for Study:2 Years to 11 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* Diagnosis of TDT as defined by: * Documented homozygous 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 mL/kilograms (kg)/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for participants initiating transfusion therapy \<24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for ≥6 months * Eligible for autologous stem cell transplant as per investigator's judgment.	* 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) * Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications * Participants with sickle cell β-thalassemia variant * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator

Inclusion Criteria

Exclusion Criteria

* Diagnosis of TDT as defined by:
* Documented homozygous 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 mL/kilograms (kg)/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for participants initiating transfusion therapy \<24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for ≥6 months
* Eligible for autologous stem cell transplant as per investigator's judgment.

* 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)
* Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
* Participants with sickle cell β-thalassemia variant
* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator

Contacts and Locations

Study Contact

Medical Information

CONTACT

617-341-6777

medicalinfo@vrtx.com

Study Locations (Sites)

SCRI at the Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203

United States

Collaborators and Investigators

Sponsor: Vertex Pharmaceuticals Incorporated

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-05-03

Study Completion Date2026-05

Study Record Updates

Study Start Date2022-05-03

Study Completion Date2026-05

Terms related to this study

Additional Relevant MeSH Terms

Beta-Thalassemia
Thalassemia
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies