ELEVATE, a Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)

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:
  GIVLAARI
• Study drug International non-proprietary name (INN) or common name:
  GIVOSIRAN
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AX16) givosiran

  givosiran
• Medical condition to be studied:
  Porphyria non-acute

  Porphyria acute
• Additional medical condition(s):
  Acute hepatic porphyria (AHP)

Population studied

• Short description of the study population:
  Study will enroll AHP patients who will be managed and treated per routine clinical practice. ; Patients will be enrolled regardless of treatment status. ; The target is to enroll 150 givosiran treated patients.
• Age groups:
  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:
  Hepatic impaired

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  To characterize the long-term real-world safety of givosiran in patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents (≥12 to <18 years of age), elderly patients (>65 years of age), and pregnant or lactating women.
• Outcomes:
  The primary outcomes of interest include the incidence of hepatic events, renal events and pancreatitis. Additional information will be collected on mortality, hospitalization and events leading to treatment discontinuation or dose adjustment. ; • The annualized rate of porphyria attacks ; • The 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version).
• Data analysis plan:
  Summary statistics will be used to characterize the study population and subgroups of interest (when appropriate). Analysis will be primarily descriptive. There are no protocol-specified hypotheses. ; Incidence rates of safety outcomes of interest will be provided during givosiran treatment as well as for patients during non-exposed periods. ; Survival analysis techniques such as Kaplan-Meier curves will be used to summarize time-to-event outcomes. ; To assess potential risk factors for time to event, Cox proportional hazards models may also be explored.