BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)

Study type

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

  Disease epidemiology

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  OXLUMO
• Study drug International non-proprietary name (INN) or common name:
  LUMASIRAN
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AX18) lumasiran

  lumasiran
• Medical condition to be studied:
  Primary hyperoxaluria
• Additional medical condition(s):
  Primary hyperoxaluria type 1 PH1

Population studied

• Short description of the study population:
  Study will enroll PH1 patients who will be managed and treated per routine clinical practice. ; Patients will be enrolled regardless of treatment status. ; The target is to enroll 100 lumasiran treated patients.
• Age groups:
  • Paediatric Population (< 18 years)

    • Neonate

      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • 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:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  200

Study design details

• Main study objective:
  The main objective of this trial is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.
• Outcomes:
  The primary outcome of interest is the incidence of adverse events in lumasiran treated patients. ; • Incidence of selected events of interest: hepatic events, kidney stones, acute kidney injury events, nephrocalcinosis, chromic kidney disease, kidney failure, and any cardiac, bone, skin, eye, hematological, or neuropathic manifestations due to oxalosis ; • 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) ; • Change in urinary oxalate excretion ; • Change in plasma oxalate
• Data analysis plan:
  Statistical analyses will be primarily descriptive in nature. Descriptive statistics for continuous variables will be reported. ; Categorical variables will be summarized as number and proportion of the relevant population, and by subgroups where appropriate. ; Summary statistics will be presented for the full study population and separately by subgroups. ; There will be no pre-defined hypotheses.