Evaluating CTX001 for treating pediatric beta-thalassemia
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
This study is testing a new treatment using gene editing to see if it can help kids with beta-thalassemia reduce or stop their need for blood transfusions.
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 | 6 sites (Nashville, Tennessee and 5 other locations) |
| Trial ID | NCT05356195 on ClinicalTrials.gov |
What this trial studies
This clinical trial is an open-label, single-dose intervention aimed at assessing the safety and efficacy of CTX001, which involves modifying a patient's own hematopoietic stem and progenitor cells using CRISPR-Cas9 technology. The study focuses on pediatric participants diagnosed with transfusion-dependent beta-thalassemia (TDT) who have a significant history of blood transfusions. Participants will undergo a conditioning regimen before receiving the modified cells, with the goal of reducing or eliminating their need for transfusions. The trial will monitor both the safety and effectiveness of this innovative treatment approach.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients diagnosed with transfusion-dependent beta-thalassemia who have a history of frequent blood transfusions.
Not a fit: Patients who have a suitable HLA-matched donor for a traditional stem cell transplant may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce or eliminate the need for blood transfusions in children with beta-thalassemia.
How similar studies have performed: Other studies using CRISPR technology for genetic diseases have shown promising results, indicating potential for success in this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion 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. Key Exclusion Criteria: * 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 Other protocol defined Inclusion/Exclusion criteria may apply.
Where this trial is running
Nashville, Tennessee and 5 other locations
- SCRI at the Children's Hospital at TriStar Centennial — Nashville, Tennessee, United States (Recruiting)
- The Hospital for Sick Children — Toronto, Canada (Recruiting)
- Universitätsklinikum Düsseldorf Hospital Duesseldorf — Düsseldorf, Germany (Recruiting)
- Ospedale Pediatrico Bambino Gesù, IRCCS — Rome, Italy (Recruiting)
- Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom (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.