An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  QALSODY
• Medicinal product name, other:
  Tofersen
• Study drug International non-proprietary name (INN) or common name:
  TOFERSEN
• Anatomical Therapeutic Chemical (ATC) code:
  (N07XX22) tofersen

  tofersen
• Medical condition to be studied:
  Amyotrophic lateral sclerosis

Population studied

• Short description of the study population:
  Participants with SOD1-ALS enrolled in TRICALS network’s Precision-ALS programme with data from participating clinical centers across multiple European countries or at ALS/MND NHC with data from participating clinical centers in the United States (tofersen users and tofersen non-users).
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)
• Estimated number of subjects:
  69

Study design details

• Main study objective:
  The primary objectives of this study are to describe demographic and clinical characteristics of participants with superoxide dismutase 1-amyotropic lateral sclerosis (SOD1-ALS); to describe the frequency of serious adverse events (SAEs) among participants with SOD1-ALS, including serious neurologic events previously reported in clinical trial participants (e.g., myelitis, radiculitis, aseptic meningitis, increased intracranial pressure, and/or papilloedema).; ; The secondary objectives of this study are to describe the frequency of new comorbid conditions, pregnancy and pregnancy outcome among participants with SOD1-ALS; and to describe the frequency of treatment discontinuation among participants with SOD1-ALS treated with tofersen.
• Outcomes:
  Baseline Demographic [Age, Participant Sex, Race/Ethnicity, Weight, Height, Body Mass Index (BMI), Family History of Amyotrophic Lateral Sclerosis (ALS)], Clinical Characteristics (Age at Diagnosis and Symptom Onset, Revised El Escorial Classification, Classification of SOD1-ALS Clinical Phenotypes, SOD1 Mutation Type, Medical History, Concomitant Medications, Disease History, Pregnancy Status), Number of Participants With SAEs, Number of Participants With New Comorbid Conditions, Number of Participants With Pregnancy and Pregnancy Outcomes, Number of Participants With Reported Treatment Discontinuation, Number of Participants With Reason for Treatment Discontinuation
• Data analysis plan:
  Data will be analysed separately for each registry network. Continuous variables will be described with summary statistics including number of observations, mean, standard deviation, median, first quartile, third quartile, and minimum and maximum values. Categorical data will be described in terms of frequencies and percentages.;