An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients with Fibrodysplasia Ossificans Progressiva (FOP) (FOPal)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  Palovarotene / Sohonos
• Anatomical Therapeutic Chemical (ATC) code:
  (M09AX11) palovarotene

  palovarotene
• Medical condition to be studied:
  Fibrodysplasia ossificans progressiva

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  70

Study design details

• Study design:
  The main aim of this study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene. This study will also describe the effectiveness of this treatment, including the effect on everyday activities and physical performance.
• Main study objective:
  To collect and assess real-world safety data in patients with FOP treated with palovarotene.
• Outcomes:
  Primary outcomes: Incidence and description of all TEAEs; of all serious and nonserious treatment-related TEAEs; of all serious TEAEs; all nonserious TEAEs; whether or not they are considered as related to the palovarotene during treatment period up to 30 days after the last dose.; ; Secondary outcomes: Use of Assistive Devices and Adaptions for Daily Living, Physical and Mental Function, investigator-reported flare-up outcomes, incidence of new bone growth, exposure.
• Data analysis plan:
  Analysis will be primarily descriptive. Descriptive summary statistics will include number of documented data, mean, standard deviation, 95% confidence intervals (CI) of the mean/median, median, minimum, maximum, or frequency counts of the data collected. Percentages will be based on the number of non missing observations. Subgroup analyses will be performed by age group, gender. Interim effectiveness and safety descriptive analyses are planned to be performed every 2 years for publication purposes as required. For the first interim analysis, a minimum of 30 participants will have to be enrolled.