A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Aug 30, 2018

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called CTX001 for people with transfusion-dependent β-thalassemia, a serious blood disorder where patients need regular blood transfusions due to their bodies not producing enough healthy hemoglobin. The trial aims to evaluate the safety and effectiveness of a special type of stem cell therapy that uses modified cells from the patient’s own blood to help their body produce normal hemoglobin.

To participate, individuals must have a confirmed diagnosis of transfusion-dependent β-thalassemia and a history of receiving a significant number of blood transfusions over the past two years. They also need to be eligible for a specific type of stem cell transplant as determined by their doctor. Throughout the trial, participants will receive a single dose of the treatment and will be monitored closely for any side effects and how well the treatment works. It’s important to know that this trial is currently active but not recruiting new participants.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by
• • 1. Documented homozygous β-thalassemia 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
• • 2. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
• • 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 allo-HSCT
• • Participants with associated α-thalassemia and \>1 alpha deletion or alpha multiplications
• • Participants 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 \<50 × 10\^9/L not related to hypersplenism
• • Other protocol defined Inclusion/Exclusion criteria may apply.