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
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Sponsor:
Amryt Pharma
Information provided by (Responsible Party):
Amryt Pharma
Tracking Information | |||||
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First Submitted Date | April 10, 2014 | ||||
First Posted Date | May 12, 2014 | ||||
Last Update Posted Date | March 12, 2024 | ||||
Actual Study Start Date | March 18, 2014 | ||||
Estimated Primary Completion Date | March 1, 2028 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures |
Hepatic Abnormalities [ Time Frame: patients will be followed for 10 years ] to evaluate the occurrence of hepatic abnormalities
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Original Primary Outcome Measures | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures |
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Original Secondary Outcome Measures |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title | LOWER: Lomitapide Observational Worldwide Evaluation Registry | ||||
Official Title | LOWER: Lomitapide Observational Worldwide Evaluation Registry | ||||
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 Type | Observational [Patient Registry] | ||||
Study Design | Observational Model: Cohort Time Perspective: Prospective |
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Target Follow-Up Duration | 10 Years | ||||
Biospecimen | Not Provided | ||||
Sampling Method | Non-Probability Sample | ||||
Study Population | Patients treated with lomitapide who agree to participate in the study. | ||||
Condition | Homozygous Familial Hypercholesterolemia | ||||
Intervention | Drug: Lomitapide
As prescribed by Physician.
Other Names:
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Study Groups/Cohorts | Lomitapide
Lomitapide as prescribed by Physician.
Intervention: Drug: Lomitapide
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Publications * | Giammanco A, Cefalu AB, Noto D, Averna MR. Therapeutic Options for Homozygous Familial Hypercholesterolemia: The Role of Lomitapide. Curr Med Chem. 2020;27(23):3773-3783. doi: 10.2174/0929867326666190121120735. | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status | Recruiting | ||||
Estimated Enrollment |
300 | ||||
Original Estimated Enrollment | Same as current | ||||
Estimated Study Completion Date | September 1, 2028 | ||||
Estimated Primary Completion Date | March 1, 2028 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria | Inclusion Criteria:
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:
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Sex/Gender |
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Ages | Child, Adult, Older Adult | ||||
Accepts Healthy Volunteers | No | ||||
Contacts |
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Listed Location Countries | Argentina, Canada, France, Greece, Italy, Netherlands, United Kingdom, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number | NCT02135705 | ||||
Other Study ID Numbers | AEGR-733-025 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product | Not Provided | ||||
IPD Sharing Statement | Not Provided | ||||
Current Responsible Party | Amryt Pharma | ||||
Original Responsible Party | Aegerion Pharmaceuticals, Inc. | ||||
Current Study Sponsor | Amryt Pharma | ||||
Original Study Sponsor | Aegerion Pharmaceuticals, Inc. | ||||
Collaborators | Not Provided | ||||
Investigators |
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PRS Account | Amryt Pharma | ||||
Verification Date | March 2024 |