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LOWER: Lomitapide Observational Worldwide Evaluation Registry (LOWER)

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ClinicalTrials.gov Identifier: NCT02135705
Recruitment Status : Recruiting
First Posted : May 12, 2014
Last Update Posted : March 12, 2024
Sponsor:
Information provided by (Responsible Party):
Amryt Pharma

Study Description
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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.

Condition or disease Intervention/treatment
Homozygous Familial Hypercholesterolemia Drug: 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 Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 10 Years
Official Title: LOWER: Lomitapide Observational Worldwide Evaluation Registry
Actual Study Start Date : March 18, 2014
Estimated Primary Completion Date : March 1, 2028
Estimated Study Completion Date : September 1, 2028

Resource links provided by the National Library of Medicine

Drug Information available for: Lomitapide

Groups and Cohorts
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Group/Cohort Intervention/treatment
Lomitapide
Lomitapide as prescribed by Physician.
 Drug: Lomitapide
As prescribed by Physician.
Other Names:
  • Juxtapid
  • Lojuxta


Outcome Measures
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Primary Outcome Measures :
  1. Hepatic Abnormalities [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of hepatic abnormalities


Secondary Outcome Measures :
  1. Gastrointestinal (GI) Events [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of GI events

  2. Tumors [ Time Frame: patients will be followed for 10 years ]
    to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)

  3. Events associated with coagulopathy [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of events associated with coagulopathy (abnormal bleeding, cerebral haemorrhage or GI bleeding)

  4. Major Adverse Cardiovascular Events (MACE) events [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of MACE events

  5. Death, including cause of death [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence and cause of death

  6. Pregnancy [ Time Frame: 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).

  7. Serum lipid levels [ Time Frame: 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.

  8. Prescriber behavior [ Time Frame: 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.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients treated with lomitapide who agree to participate in the study.
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..
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02135705


Contacts
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Contact: Janet Boylan 1 855 303 2347 Janet.Boylan@AmrytPharma.com

Locations
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Sponsors and Collaborators
Amryt Pharma
Investigators
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Study Director: Janet Boylan Amryt Pharmaceuticals
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Amryt Pharma
ClinicalTrials.gov Identifier: NCT02135705    
Other Study ID Numbers: AEGR-733-025
First Posted: May 12, 2014    Key Record Dates
Last Update Posted: March 12, 2024
Last Verified: March 2024
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Homozygous Familial Hypercholesterolemia
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
 Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias