Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Jul 26, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called CTX001 for people with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The treatment involves modifying a patient’s own blood stem cells using a technique called CRISPR-Cas9, which helps to correct the genetic issues causing these blood disorders. The goal is to see how safe and effective a single dose of this treatment is for participants who require regular blood transfusions or experience severe pain episodes due to their conditions.

To be eligible for the trial, participants must have a confirmed diagnosis of TDT or severe SCD and be suitable for a specific type of stem cell transplant. For TDT patients, this means having a history of needing a certain amount of blood transfusions each year, while SCD patients should have experienced at least two severe pain episodes annually. Participants will receive the modified cells and will be monitored closely to assess how well the treatment works and if there are any side effects. This study is currently recruiting and welcomes individuals of all genders between the ages of approximately 4 to 12 years.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• * Participants with TDT and SCD:
• • Eligible for autologous stem cell transplant as per investigator's judgment.
• * Participants with TDT:
• * Diagnosis of TDT as defined by:
• • 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
• • History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
• * Participants with SCD:
• * 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
• Key Exclusion Criteria:
• * Participants with TDT and SCD:
• • 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)
• • Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
• * Participants with TDT:
• • Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
• • Participants with sickle cell β-thalassemia variant
• * Participants with SCD:
• • History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening
• • Other protocol defined Inclusion/Exclusion criteria may apply.