Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Description

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

Conditions

Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases

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Study Overview

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Study Details

Study overview

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

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Condition
Sickle Cell Disease
Intervention / Treatment

-

Contacts and Locations

Charlotte

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

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Memphis

St. Jude Children's Research Hospital, Memphis, Tennessee, United States, 38105

Nashville

The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, 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

  • * 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
  • * Hydroxyurea (HU) failure unless HU intolerant
  • * Eligible for autologous stem cell transplant as per investigators judgment
  • * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor
  • * Prior hematopoietic stem cell transplant (HSCT).
  • * Clinically significant and active bacterial, viral, fungal, or parasitic infection

Ages Eligible for Study

2 Years to 11 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Vertex Pharmaceuticals Incorporated,

Study Record Dates

2026-05