Homozygous Familial Hypercholesterolemia
Conditions
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.
Interventions
DRUGLomitapide
As prescribed by Physician.
Sponsors
Amryt Pharma
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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..
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hepatic Abnormalities | patients will be followed for 10 years | to evaluate the occurrence of hepatic abnormalities |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Argentina, Canada, France, Greece, Italy, Netherlands, United Kingdom, United States
Contacts
STUDY_DIRECTORJanet Boylan
Amryt Pharmaceuticals
Outcome results
None listed