A Registry of Patients Treated with Fintepla (TAPESTRY Registry)

Study type

• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (N03AX26) fenfluramine

  fenfluramine

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  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:
  600

Study design details

• Main study objective:
  Assess the long-term cardiac safety of fenfluramine as prescribed in routine clinical practice for fenfluramine approved indications, with a focus on (1) Incidence of valvular heart disease (VHD) and (2) Incidence of pulmonary arterial hypertension (PAH)
• Outcomes:
  The primary outcome variables for all patients will include but not be limited to: 1) Date of ECHO 2) Grade (absent/trace, mild, moderate, severe) of valve regurgitation and affected valves 3) Presence or absence of elevated PASP (equal to or more than 35 mm Hg) 4) Valvular or any other abnormality reported on baseline ECHO. They are further described in protocol section 9.3.1. The secondary outcome variables are related to growth retardation and echocardiographic monitoring characteristics. They are further described in protocol section 9.3.2.
• Data analysis plan:
  The analyses will be descriptive. Continuous variables will be described by the number of valid cases and missing data, mean, standard deviation, median, first quartile, third quartile, minimum, and maximum. Categorical variables will be described as the total number and relative percentage per category. 95% confidence intervals (CIs) will be calculated when relevant. There will be no imputation of missing data. Incidence rates will be calculated by dividing the number of new cases (up to the first event) of VHD or PAH by the number of person-years at risk.