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

Argentina
Austria
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

Klinikum der Universitaet Muenchen-DEU;
Umberto I' Hospital-ITA;
Azienda Ospedaliera Universitaria â œPoliclinico Paolo Giacconeâ di Palermo-ITA;
Azienda Ospedaliero Universitaria Policlinico di Bari-ITA;
CNR - Regione Toscana-ITA;
Azienda Sanitaria Ospedaliera San Luigi Gonzaga-ITA;
Ospedale Monaldi-ITA;
Azienda Ospedaliera Universitaria Integrata di Verona-ITA;
Policlinico Federico II-ITA;
Dipartimento Clinica E Terapia Medica Applicata -ITA;
Azienda Ospedaliera "G.Brotzu"-ITA;
Azienda Ospedaliera Universitaria Padova -ITA;
Policlinico S. Orsola-Malpighi-ITA;
Università degli Studi di Genova/Policlinico S. Martino-ITA;
Ospedale Bassini-ITA;
Azienda Ospedaliero-Universitaria - Mater Domini-ITA;
Azienda Ospedaliero-Universitaria S. Anna di Ferrara-ITA;
Centre Hospitalier Universitaire Strasbourg-FRA;
Centre Hospitalier Regional Universitaire de Lille-FRA;
Hopital Louis Pradel-FRA;
Hopital de la Pitie-Salpetriere-FRA

Contact details

Beatriz Borredá

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
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)