Status:
RECRUITING
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborating Sponsors:
CRISPR Therapeutics
Conditions:
Beta-Thalassemia
Thalassemia
Eligibility:
All Genders
2-11 years
Phase:
PHASE3
Brief Summary
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 progen...
Eligibility Criteria
Inclusion
- Key
- 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
- 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.
Key Trial Info
Start Date :
May 3 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 31 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT05356195
Start Date
May 3 2022
End Date
May 31 2026
Last Update
October 14 2025
Active Locations (6)
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1
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States, 37203
2
Hospital for Sick Children - Hematology
Toronto, Canada
3
University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
4
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy