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Registry to evaluate the long-term safety and effectiveness of lomitapide

LOWER: Lomitapide Observational Worldwide Evaluation Registry

Amryt Pharma · NCT02135705

This study is tracking how safe and effective the drug lomitapide is for people of all ages with Homozygous Familial Hypercholesterolemia over the long term.

Quick facts

Study typeObservational
Enrollment300 (estimated)
SexAll
SponsorAmryt Pharma (industry)
Locations75 sites (Birmingham, Alabama and 74 other locations)
Trial IDNCT02135705 on ClinicalTrials.gov

What this trial studies

This global observational registry aims to assess the long-term safety and effectiveness of lomitapide in patients with Homozygous Familial Hypercholesterolemia. It involves a multicenter approach where patients of all ages who have started treatment with lomitapide will be monitored for adverse events and adherence to screening recommendations. The study will collect data on patient outcomes over time to better understand the drug's impact in real-world settings.

Who should consider this trial

Good fit: Ideal candidates include patients of all ages who have initiated treatment with lomitapide and can provide informed consent.

Not a fit: Patients currently participating in clinical trials or receiving lomitapide through compassionate use may not benefit from this registry.

Why it matters

Potential benefit: If successful, this registry could provide valuable insights into the long-term safety and effectiveness of lomitapide, potentially improving treatment strategies for patients with high cholesterol levels.

How similar studies have performed: Other observational studies have shown success in evaluating long-term treatment effects, suggesting that this approach is viable.

Eligibility criteria

Show full inclusion / exclusion criteria 
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..

Where this trial is running

Birmingham, Alabama and 74 other locations

+25 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Homozygous Familial Hypercholesterolemia

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.