LOWER: Lomitapide Observational Worldwide Evaluation Registry (AEGR-733-025)

Study identification

• EU PAS number:
  EUPAS5326
• Study ID:
  47071
• Official title and acronym:
  LOWER: Lomitapide Observational Worldwide Evaluation Registry (AEGR-733-025)
• DARWIN EU® study:
  No
• Study countries:
  Canada

  France

  Greece

  Italy

  Netherlands

  United Kingdom

  United States
• Study description:
  The registry is designed to evaluate the long-term safety and effectiveness of lomitapide in clinical practice. The objectives of the registry are: ; • To evaluate the occurrence of the following in patients treated with lomitapide: Hepatic abnormalities, Gastrointestinal (GI) events, Small bowel, hepatic, colorectal and pancreatic tumours, Events associated with coagulopathy, Major Adverse Cardiovascular Events (MACE) events, Death, including cause of death, ; • To evaluate the occurrence and outcomes of pregnancy in females of reproductive potential treated with lomitapide, with or without consultation with a teratologist and regardless of the outcome of the pregnancy (live birth, elective or spontaneous abortion or still birth). The outcomes of primary interest are major congenital anomalies. Refer to Section 7.2.3 for more detail., ; • To evaluate the long-term effectiveness of lomitapide in maintaining control of serum lipid levels in clinical practice., ; • To evaluate whether prescribers of lomitapide enroled at registry sites are following the screening and monitoring recommendations as specified in the product information (PI) and the prescriber educational materials aimed at risk minimisation.
• Study status:
  Ongoing

Research institutions and networks

Institutions

CHU Conception - FRA;

Metropolitan Hospital

Radboud university medical center (Radboudumc)

Netherlands

Assitance Publique des Hopitaux de Marseille (APHM)

France

Erasmus Medical Centre Rotterdam

Umberto I' Hospital-ITA;
CNR - Regione Toscana-ITA;
Ospedale Monaldi-ITA;
Dipartimento Clinica E Terapia Medica Applicata -ITA;
Hopital Louis Pradel-FRA;
Hopital de la Pitie-Salpetriere-FRA
FRA – CHU Conception
ITA- Azienda Ospedaliera Universitaria Federico II
ITA- Azienda Ospedaliero-Universitaria Mater Domini Catanzaro
FRA- CHU Lyon
FRA-CHU - l'Assistance Publique - Hôpitaux de Marseille
FRA-AP-HP - HU La Pitié-Salpêtrière – Charles-Foix
GRC- University General Hospital of Heraklion
GRC- Metropolitan Hospital

Study timelines

Date when funding contract was signed

Planned:02/12/2013

Actual:20/12/2013

Study start date

Planned:01/03/2014

Actual:18/03/2014

Data analysis start date

Planned:18/03/2028

Date of interim report, if expected

Planned:01/07/2025

Date of final study report

Planned:13/07/2028

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Amryt, DAC

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)