LOWER: Lomitapide Observational Worldwide Evaluation Registry

Amryt Pharma

Status

Enrolling

Conditions

Homozygous Familial Hypercholesterolemia

Treatments

Drug: Lomitapide

Study type

Observational

Funder types

Industry

Identifiers

NCT02135705

AEGR-733-025

Details and patient eligibility

About

This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.

Full description

To evaluate the occurrence of adverse events of special interest, long term effectiveness of lomitapide, and to evaluate whether prescribers of lomitapide are following screening and monitoring recommendations as specified in product labeling.

Enrollment

300 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

Patients of all ages, including minors, who have initiated commercial treatment with lomitapide prior to or at time of registry enrolment.
Patients who have the ability to understand the requirements of the study and provide written informed consent to comply with the study data collection procedures or paediatric patients with the consent of a parent or legal guardian.

Patients ≥7 years of age (or above the age determined by the IRB/EC and in accordance with the local regulations and requirements) must also provide written informed assent forms.

Exclusion criteria

Patients who are receiving lomitapide in clinical trials or through compassionate use, where patients are followed under a separate protocol.
Patients receiving an investigational agent, defined as any drug or biologic agent other than lomitapide that has not received market authorization in the country of participation, at time of lomitapide initiation and continuing to receive an investigational agent at time of registry enrolment. These patients may be enrolled if receiving lomitapide when the investigational agent is discontinued..