European Post-Authorization Registry for RAVICTI® (glycerol phenylbutyrate) Oral Liquid in Partnership with the European Registry and Network for Intoxication Type Metabolic Diseases (E-IMD) (HZNP-RAV-401)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study

Non-interventional study

• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary and secondary data collection
• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Multi-centre, prospective, PASS study

Study drug and medical condition

• Name of medicine:
  RAVICTI
• Study drug International non-proprietary name (INN) or common name:
  GLYCEROL PHENYLBUTYRATE
• Anatomical Therapeutic Chemical (ATC) code:
  100000144759

  glycerol phenylbutyrate
• Medical condition to be studied:
  Urea cycle disorder

Population studied

• Short description of the study population:
  The study population included adult and pediatric patients diagnosed with urea cycle disorder received treatment with RAVICTI or alternative nitrogen scavenging medication.
• 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)
• Special population of interest:
  Other

  Pregnant women

  Renal impaired
• Special population of interest, other:
  Patients with urea cycle disorder
• Estimated number of subjects:
  200

Study design details

• Main study objective:
  Evaluation and characterization of the safety profile of RAVICTI and long-term outcomes in UCD patients treated with RAVICTI. The registry includes a comparator group treated with alternative nitrogen scavenging medication. In all other relevant characteristics (age group, severity of the UCD and ge
• Outcomes:
  Collect relevant long-term safety data in patients with UCDs treated with RAVICTI and also collect and compare this data to a comparator group treated with alternative nitrogen scavenging medication., For the RAVICTI and comparator group, collect and compare information on: Incidence rate and type of cancer Occurrence of potential PAA (phenylacetate) toxicity Safety information in patients with concurrent renal impairment Pregnancy outcomes in children born to female patients
• Data analysis plan:
  Descriptive statistics including number of observations, mean, standard deviation (SD), median, minimum, and maximum for continuous variables; and n and percent for categorical variables. Data will be presented for patients in the RAVICTI and the comparator group. Data of comparator group patients will only be presented if consent is given. Additional subgroups may be examined, as appropriate (pediatric versus adult, UCD type, etc.). Disposition data will be summarized with descriptive statistics. Demographic and clinical data will be summarized with descriptive statistics. Post-baseline values and/or change from baseline in the outcome variables will be summarized with descriptive statistics, and, where appropriate, graphical presentations. Differences in outcome variables between the groups will be evaluated using generalized linear mixed models (continuous and dichotomous endpoints) as well as hazard models for dichotomous and, when adequate, multinomial endpoints.

Documents

Results tables

HZNP-RAV-401_Synopsis