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 identification

• PURI:
  https://redirect.ema.europa.eu/resource/47168
• EU PAS number:
  EUPAS32190
• Study ID:
  47168
• Official title and acronym:
  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)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Belgium

  Bulgaria

  Czechia

  Denmark

  France

  Germany

  Hungary

  Ireland

  Israel

  Italy

  Latvia

  Netherlands

  Norway

  Portugal

  Romania

  Slovakia

  Slovenia

  Spain

  Sweden

  Switzerland

  United Kingdom

  United Kingdom (Northern Ireland)
• Study description:
  This is a 10-year prospective cohort study using data collected in a European disease registry for XLH. The PASS is non-interventional so all data collected will arise from the usual clinical management of these patients. ; ; Primary objectives ; 1. To evaluate the frequency and severity of safety outcomes in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease and adults, and who are treated with burosumab for XLH, including but not limited to: long term safety (as evidenced by death, hospitalisations, cardiovascular disease, cancer all sites), hyperphosphataemia and its complications, ectopic mineralisation, increased parathyroid hormone levels. ; 2. To prospectively evaluate the frequency and outcomes of pregnancies in female subjects treated with burosumab. ; 3. To prospectively evaluate the frequency and severity of safety outcomes in subjects with mild to moderate chronic kidney disease at baseline treated with burosumab. ; ; Secondary objectives: ; 1. To perform a retrospective cohort analysis using data from the XLH Registry to compare the safety outcomes in subjects exposed to burosumab to those in subjects receiving alternative treatments for XLH.
• Study status:
  Ongoing

Research institution and networks

Institutions

Karolinska Institutet

Sweden

Study timelines

Date when funding contract was signed

Actual: 20/11/2018

Study start date

Planned: 24/04/2019

Actual: 24/04/2019

Date of interim report, if expected

Planned: 26/10/2020

Actual: 26/10/2021

Date of final study report

Planned: 31/12/2028

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Kyowa Kirin International plc.

Study protocol

Initial protocol

XLH Registry Protocol Amendment 2_Final_15Feb2019.pdf

Updated protocol

XLH Registry Protocol Amendment 3 with embedded PASS_27Oct2021_clean.pdf

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)