A Registry of Patients with Biallelic Pro Opiomelanocortin (POMC), Proprotein Convertase Subtilisin/Kexin Type 1 (PCSK1), or Leptin Receptor (LEPR) Deficiency Obesity, or Bardet-Biedl Syndrome (BBS), Treated with Setmelanotide

Study type

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

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  SETMELANOTIDE
• Medical condition to be studied:
  Pro-opiomelanocortin deficiency

  Leptin receptor deficiency

  Laurence-Moon-Bardet-Biedl syndrome
• Additional medical condition(s):
  Proprotein convertase subtilisin/kexin type 1 deficiency

Population studied

• Age groups:
  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:
  50

Study design details

• Main study objective:
  To assess the long-term safety of setmelanotide as prescribed in routine practice for patients with biallelic POMC/PCSK1 or LEPR deficiency obesity, or BBS according to the current local prescribing information.
• Outcomes:
  The primary outcome measure for this study is the occurrence (including seriousness and relatedness) of all AEs in patients with biallelic POMC/PCSK1 or LEPR deficiency obesity, or BBS, treated with setmelanotide. •The occurrence and characterisation of AESIs (prolonged penile erection and depression). •The occurrence and characterisation of all AEs and AESIs in patients with hepatic impairment, severe renal impairment, and pregnant/breastfeeding women. •Long-term weight change after initiation of setmelanotide •The characterisation of biallelic POMC/PCSK1 or LEPR deficiency obesity, or BBS
• Data analysis plan:
  Primary outcomes will be summarised as frequency (counts and percentages) and incidence rates for the overall population, and for new (incident), current (prevalent), or past setmelanotide users, separately. Incidence rates will be calculated by dividing the number of AEs by the number of person-years at risk. Adverse events/AESIs will be summarised using counts and percentages for the entire study population and for new (incident), current (prevalent), and past users of setmelanotide, separately. To assess setmelanotide use (past vs. current vs. new users) with baseline demographic and disease-related characteristics, a multivariable logistic regression model will be fitted against all explanatory variables, including baseline demographics and disease-related characteristics. All secondary outcomes will be summarised as frequency (counts and percentages) and incidence rates for the overall population and for new (incident), current (prevalent), and past users of setmelanotide.