LOWER: Lomitapide Observational Worldwide Evaluation Registry

Recruiting

• Conditions: Homozygous Familial Hypercholesterolemia
• Interventions: Drug: Lomitapide

• Registration Number: NCT02135705
• Lead Sponsor: Amryt Pharma

Brief Summary

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.

Detailed 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.

Study Timeline

• Study Start: 2014-03-18
• Study First Submitted: 2014-04-10
• Study First Submitted QC: 2014-05-08
• Study First Posted: 2014-05-12
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-11
• Last Update Posted: 2024-03-12
• Primary Completion: 2028-03-01
• Study Completion: 2028-09-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

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..

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Lomitapide	Lomitapide	Lomitapide as prescribed by Physician.

Primary Outcome Measures

Name	Time	Method
Hepatic Abnormalities	patients will be followed for 10 years	to evaluate the occurrence of hepatic abnormalities

Secondary Outcome Measures

Name	Time	Method
Gastrointestinal (GI) Events	patients will be followed for 10 years	to evaluate the occurrence of GI events
Tumors	patients will be followed for 10 years	to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)
Events associated with coagulopathy	patients will be followed for 10 years	to evaluate the occurrence of events associated with coagulopathy (abnormal bleeding, cerebral haemorrhage or GI bleeding)
Major Adverse Cardiovascular Events (MACE) events	patients will be followed for 10 years	to evaluate the occurrence of MACE events
Death, including cause of death	patients will be followed for 10 years	to evaluate the occurrence and cause of death
Pregnancy	patients will be followed for 10 years	to evaluate the occurrence and outcomes of pregnancy in females of reproductive potential treated with lomitapide. Patients who become pregnant will be offered enrolment into a separate Pregnancy Exposure Registry (PER).
Serum lipid levels	patients will be followed for 10 years	to evaluate the long-term effectiveness of lomitapide in maintaining control of serum lipid levels in a clinical practice setting.
Prescriber behavior	patients will be followed for 10 years	to evaluate whether prescribers of lomitapide enrolled at registry sites are following the screening and monitoring recommendations as specified in the PI and the prescriber educational materials aimed at risk minimization.

Trial Locations

Locations (74)

Brookwood Baptist Medical Center; 🇺🇸 Birmingham, Alabama, United States

Springhill Physician Practices; 🇺🇸 Mobile, Alabama, United States

Northern Arizona Healthcare; 🇺🇸 Cottonwood, Arizona, United States

One Medical; 🇺🇸 Gilbert, Arizona, United States

Scottsdale Family Health; 🇺🇸 Phoenix, Arizona, United States

Pima Heart; 🇺🇸 Tucson, Arizona, United States

Arkansas Heart Center; 🇺🇸 Fort Smith, Arkansas, United States

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

University of Colorado Health; 🇺🇸 Aurora, Colorado, United States

Alfieri Cardiology; 🇺🇸 Newark, Delaware, United States

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