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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 : December 4, 2020
Sponsor:
Information provided by (Responsible Party):
Amryt Pharma

Tracking Information
First Submitted Date April 10, 2014
First Posted Date May 12, 2014
Last Update Posted Date December 4, 2020
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
 (submitted: May 8, 2014)
Hepatic Abnormalities [ Time Frame: patients will be followed for 10 years ]
to evaluate the occurrence of hepatic abnormalities
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: December 2, 2020)
  • Gastrointestinal (GI) Events [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of GI events
  • Tumors [ Time Frame: patients will be followed for 10 years ]
    to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)
  • 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)
  • Major Adverse Cardiovascular Events (MACE) events [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of MACE events
  • Death, including cause of death [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence and cause of death
  • 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).
  • 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.
  • 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.
Original Secondary Outcome Measures
 (submitted: May 8, 2014)
  • Gastrointestinal (GI) Events [ Time Frame: patients will be followed for 10 years ]
    to evaluate the occurrence of GI events
  • Tumors [ Time Frame: patients will be followed for 10 years ]
    to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)
  • 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)
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
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Adult patients treated with lomitapide who agree to participate in the study.
Condition Homozygous Familial Hypercholesterolemia
Intervention Drug: Lomitapide
As prescribed by Physician.
Other Names:
  • Juxtapid
  • Lojuxta
Study Groups/Cohorts Lomitapide
Lomitapide as prescribed by Physician.
Intervention: Drug: Lomitapide
Publications * Giammanco A, Cefalù 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. Review.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: May 8, 2014)
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:

  • Adult patients (age ≥18 years) who meet one of the following two criteria:

Initiating treatment with lomitapide at the time of registry enrolment, or initiated treatment with lomitapide within 36 months prior to enrolment into the registry and after lomitapide commercial availability in the country.

  • 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.

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 MA in the country of participation.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Sallyann O'Brien 1 855 303 2347 medinfo@amrytpharma.com
Listed Location Countries 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
Responsible Party Amryt Pharma
Study Sponsor Amryt Pharma
Collaborators Not Provided
Investigators
Study Director: Sallyann O'Brien Amryt Pharmaceuticals
PRS Account Amryt Pharma
Verification Date December 2020