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 identification

• PURI:
  https://redirect.ema.europa.eu/resource/48823
• EU PAS number:
  EUPAS48822
• Study ID:
  48823
• Official title and acronym:
  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
• DARWIN EU® study:
  No
• Study countries:
  France

  Germany

  Italy

  Netherlands

  Spain

  United Kingdom

  United States
• Study description:
  To collect real-world, long-term safety data for the use of setmelanotide in patients with biallelic (homozygous or compound heterozygous) pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency obesity, or Bardet-Biedl Syndrome (BBS). The primary objective of this study is to assess the long-term safety of setmelanotide as prescribed in routine practice for patients with biallelic (homozygous or compound heterozygous) POMC/PCSK1, or LEPR deficiency obesity, or BBS, according to the current local prescribing information. The secondary objectives of this study are to document and characterise adverse events of special interest (AESIs), including prolonged penile erections and depression (including suicidal ideation) for all patients, to document and characterise AESIs and adverse events (AEs) in special populations (including patients with hepatic impairment, patients with severe renal impairment, and those who become pregnant or are breastfeeding), to evaluate long-term effectiveness of setmelanotide as prescribed in routine clinical practice, and to describe baseline characteristics and history of obesity in patients treated with setmelanotide. Exploratory objectives are to document any cases of melanoma and their characteristics, and to document obesity-related hospitalisations and surgeries.
• Study status:
  Planned

Research institution and networks

Institutions

Evidera

United Kingdom

Study timelines

Date when funding contract was signed

Planned: 18/08/2021

Actual: 18/08/2021

Study start date

Planned: 31/03/2023

Date of final study report

Planned: 30/09/2032

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Rhythm Pharmaceuticals

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)