Evaluating CTX001 for treating severe sickle cell disease in children
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
This study is testing a one-time treatment using a new gene-editing method to see if it can help children with severe sickle cell disease who haven't responded to other medications.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 2 Years to 11 Years |
| Sex | All |
| Sponsor | Vertex Pharmaceuticals Incorporated Industry-sponsored |
| Locations | 7 sites (Charlotte, North Carolina and 6 other locations) |
| Trial ID | NCT05329649 on ClinicalTrials.gov |
What this trial studies
This interventional phase 3 trial assesses the safety and efficacy of a single-dose treatment using CTX001, which involves modifying a patient's own hematopoietic stem and progenitor cells with CRISPR-Cas9 technology. The study focuses on pediatric participants diagnosed with severe sickle cell disease who have either failed hydroxyurea treatment or are intolerant to it. Participants will be monitored for adverse effects and treatment outcomes over the course of the study.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients with severe sickle cell disease who have experienced hydroxyurea failure or intolerance.
Not a fit: Patients with a willing and healthy 10/10 HLA-matched related donor or those who have previously undergone hematopoietic stem cell transplant may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life and health outcomes for children suffering from severe sickle cell disease.
How similar studies have performed: Other studies utilizing CRISPR technology for genetic disorders have shown promising results, indicating potential for success in this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion 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 Key Exclusion Criteria: * 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 Other protocol defined Inclusion/Exclusion criteria may apply.
Where this trial is running
Charlotte, North Carolina and 6 other locations
- Atrium Health Levine Children's Hospital — Charlotte, North Carolina, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)
- The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers — Nashville, Tennessee, United States (Recruiting)
- University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology — Dusseldorf, Germany (Recruiting)
- Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS — Rome, Italy (Recruiting)
- St Mary's Hospital — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Medical Information
- Email: medicalinfo@vrtx.com
- Phone: 617-341-6777
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.