REG-501: A Registry of Patients with β-Thalassemia Treated with Betibeglogene Autotemcel

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  ZYNTEGLO
• Medical condition to be studied:
  Thalassaemia beta

Population studied

• Age groups:
  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  8

Study design details

• Main study objective:
  Evaluate the long-term and short-term safety of treatment with betibeglogene autotemcel (beti-cel) in patients with β-thalassaemia, including the risk of newly diagnosed malignancies. Evaluate the long-term effectiveness of treatment with betibeglogene autotemcel (beti-cel).
• Outcomes:
  Incidence of Adverse Events of Interest (AEIs) through 15 years post-betibeglogene autotemcel (beti-cel) infusion, • Incidence of Serious Adverse Events (SAE) through 15 years post-beti-cel infusion (regardless of relatedness to beti-cel) • Incidence of beti-cel-related Adverse Events (AEs) through 15 years post-beti-cel infusion • Event-free survival through 15 years post-beti-cel infusion • Transfusion independence through 15 years post-beti-cel infusion
• Data analysis plan:
  Descriptive analyses will be performed to gain an understanding of the qualitative and quantitative nature of the data collected and the characteristics of the patient population. Formal statistical hypothesis testing will not be performed. Continuous variables will be reported as mean (and standard deviation), or median (and minimum, maximum) where appropriate. Categorical variables will be summarized as number and proportion of the total study population.