A Study to Identify and Characterize LAL-D Patients in High-risk Populations
NCT ID: NCT02345421
Last Updated: 2016-05-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
640 participants
OBSERVATIONAL
2014-12-31
2015-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
Study Groups
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At risk population
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
2. Non-obese\*\* patients with low high-density lipoprotein (HDL)
3. Non-obese\*\* patients with unexplained and persistently elevated liver transaminases,
4. Non-obese\*\* patients with hepatomegaly
5. Patients with cryptogenic cirrhosis
6. Patients with biopsy-proven microvesicular or mixed micro/macrovesicular steatosis without a known etiology
7. Patients with presumed Familial Hypercholesterolemia (FH) in which genetic analysis was performed for the genes encoding the low-density lipoprotein receptor (LDLR), Apo-B and PCSK9 genes and no disease-causing mutations were identified
8. Patients with presumed FH with unclear family history
9. Patients with autosomal recessive hypercholesterolemia (other than homozygous FH)
10. Patients with autosomal recessive low HDL of unknown etiology
Also, patient must meet the following:
* Patient or patient's parent or legal guardian (if applicable) consents to participate in the study and provides informed consent prior to any study procedures being performed. If the patient is of minor age; he/she is willing to provide assent where required per local regulations, and if deemed able to do so.
* Patient is willing and able to comply with protocol requirements.
* Patients who do not fall into one of the aforementioned categories (cohorts) but are considered highly suspicious for LAL D should be tested to rule out the disorder outside of the study at the discretion of the Investigator.
Exclusion Criteria
* Other confirmed genetic liver diseases (e.g., Wilson's disease, hemochromatosis, alpha 1-antitrypsin).
2 Years
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Birmingham, Alabama, United States
Phoenix, Arizona, United States
Los Angeles, California, United States
San Francisco, California, United States
Gainesville, Florida, United States
Jacksonville, Florida, United States
Miami, Florida, United States
New Orleans, Louisiana, United States
Minneapolis, Minnesota, United States
The Bronx, New York, United States
Durham, North Carolina, United States
Oklahoma City, Oklahoma, United States
Portland, Oregon, United States
Houston, Texas, United States
San Antonio, Texas, United States
Countries
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Other Identifiers
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LAL-CSS01
Identifier Type: -
Identifier Source: org_study_id
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