Kuvan® Adult Maternal Paediatric European Registry (KAMPER)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational, multicentre, drug registry study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  SAPROPTERIN DIHYDROCHLORIDE
• Medical condition to be studied:
  Phenylketonuria

  Tetrahydrobiopterin deficiency

Population studied

• Short description of the study population:
  Subjects treated with Kuvan®.; The following inclusion and exclusion criteria must have been fulfilled:; Inclusion criteria; • Adult or paediatric patients (no age limit) of either gender with HPA due to PKU or BH4 deficiency,; • Have been shown to be responsive to BH4 or Kuvan,; • Currently being treated with Kuvan at a participating site,; • Patient or parent/legal guardian willing and able to provide written, signed informed consent and to give consent before any data collection. If a child is old enough to read and write, a separate assent form was given.; Exclusion criteria; • Known hypersensitivity to Kuvan,; • Legal incapacity or limited legal capacity without legal guardian representation,; • Breast-feeding.
• 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:
  Renal impaired

  Hepatic impaired

  Pregnant women
• Estimated number of subjects:
  625

Study design details

• Main study objective:
  The primary objective is to assess the long-term safety in subjects treated with Kuvan
• Outcomes:
  The primary endpoint: Incidence and description of Adverse events and Serious Adverse Events (AE/SAEs), Secondary endpoints: Incidence of AEs/SAEs in specific populations elderly (65 years), children and subjects with renal or hepatic insufficiency. Description of somatic growth in BH4 deficient children <3 years, Neurocognitive outcomes, Neurological and psychiatric assessment, Diet and Kuvan treatment adherence, Long-term sensitivity to Kuvan treatment, Pregnancy outcomes and delivery outcomes.
• Data analysis plan:
  Interim analyses will be performed annually. Each interim analysis will include cross-sectional analyses of data regarding the safety and other outcomes such as clinical parameters, laboratory parameters or neurocognitive outcomes. For each variable, descriptive statistics from the current and previous annual analysis will be included in the interim report. Analyses will be descriptive. Confidence intervals will be provided for the primary endpoints and for secondary endpoints when relevant.

Documents

Study results

Abstract KAMPER_Final CSR_final v1.0_10Dec2021_Redacted