Non-interventional Post-Authorisation Safety Study of Burosumab in the Treatment of Children >1 year of age, Adolescents and Adults with X-Linked Hypophosphataemia (XLH PASS)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  CRYSVITA
• Study drug International non-proprietary name (INN) or common name:
  BUROSUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  100000097124

  Other drugs affecting bone structure and mineralization

  200000003181

  burosumab
• Additional medical condition(s):
  X-Linked Hypophosphataemia (XLH)

Population studied

• Age groups:
  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:
  Pregnant women
• Estimated number of subjects:
  400

Study design details

• Main study objective:
  The purpose of the study is to characterise the treatment, progression and long-term outcomes of XLH. The safety outcomes will include: ; • Long term safety: Death, Hospitalisations, Cardiovascular disease, Cancer (all sites) ; • Hyperphosphataemia ; • Ectopic mineralisation ; • Increased parathyroid hormone levels; • Effects in patients with mild to moderate chronic kidney disease at baseline
• Outcomes:
  1. Evaluate frequency & severity of safety outcomes in Children & Adults with XLH with radiographic evidence of bone disease, who are treated with burosumab ; 2. Prospectively evaluate frequency & outcomes of pregnancies in female subjects treated with burosumab ; 3. Prospectively evaluate frequency & severity of safety outcomes in patients with mild to moderate CKD treated with burosumab; To perform a retrospective cohort analysis using data from the XLH Registry to compare the safety outcomes in subjects treated with burosumab to those outcomes in subjects receiving alternative treatments for XLH
• Data analysis plan:
  Given the orphan indication and likely relatively small number of burosumab subjects (estimated to be approximately 400 subjects in the XLH Registry at the end of 10 years) the data analysis will be in the form of descriptive statistics as the sample size will not be sufficient for formal comparative analysis. Medical history and drug details will be captured in the XLH Registry via use of ICD10 codes and the World Health Organization Drug Dictionary (WHODD). Adverse events will be coded using the latest version of the Medical Dictionary for Regulatory Activities (MedDRA).

Documents

Study, other information

PASS Annual Progress Report 2019

PASS Annual Progress Report 2020

PASS Interim Study Report 4Oct2021

PASS Annual Progress Report 2022

PASS Annual Progress Report 2023

PASS Annual Progress Report 2024.pdf