EUCTR2021-002172-39-DE
Active, not recruiting
Phase 1
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent ß-Thalassemia
ConditionsTransfusion-Dependent ß ThalassemiaMedDRA version: 20.1Level: LLTClassification code 10054660Term: Thalassemia betaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Transfusion-Dependent ß Thalassemia
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Enrollment
- 15
- Status
- Active, not recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subjects 2 through 11 years of age, inclusive, on the date of informed consent.
- •Diagnosis of transfusion\-dependent ß\-thalassemia (TDT) as defined by:
- •a. Documented homozygous or compound heterozygous ß\-thalassemia including ß\-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before start of busulfan conditioning.
- •b. History of at least 100 mL/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 subjects 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.
- •Other protocol defined inclusion criteria may apply.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1\.2 Adults (18\-64 years) no
- •F.1\.2\.1 Number of subjects for this age range
Exclusion Criteria
- •An available 10/10 Human Leukocyte Antigen (HLA)\-matched related donor.
- •Prior allo\-HSCT.
- •Subjects with associated a\-thalassemia and \>1 alpha chain deletion or alpha multiplications.
- •Subjects with sickle cell beta thalassemia variant.
- •Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
- •White blood cell (WBC) count \<3 × 10^9/L or platelet count \<150 × 10^9/L not related to hypersplenism.
- •Other protocol defined exclusion criteria may apply.
Outcomes
Primary Outcomes
Not specified
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