Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01556906
• Date of registration: 07/03/2012
• Prospective Registration: No
• Primary sponsor: Aegerion Pharmaceuticals, Inc.
• Public title: Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor
• Scientific title: A Phase II Open Label, Dose-Escalation Study to Determine the Safety, Tolerability and Efficacy of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor BMS-201038 in Patients With Homozygous Familial Hypercholeterolemia
• Date of first enrolment: June 2003
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01556906
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Dan J Rader, MD
• Affiliation: University of Pennsylvania

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Males and females =13 years of age

2. Clinical diagnosis of HoFH AND one of the following (a, b, or c):

- Documented functional mutation in both LDL receptor alleles, OR

- Skin fibroblast LDL receptor activity <20% of normal, OR

- TC >500 mg/dL AND triglycerides < 300 mg/dL AND both parents with documented TC
>250 mg/dL

3. Body weight =40 kg

4. Negative screening pregnancy test if female of child-bearing potential

5. Subjects must be willing and able to comply with all study-related procedures

6. Subjects must be willing and able to go off all lipid-lowering medications, dietary
supplements (psyllium preparations) and LDL apheresis within 4 weeks prior to the
Baseline visit until the end of the study.

Exclusion Criteria:

1. Uncontrolled hypertension defined as: systolic blood pressure >180 mmHg, diastolic
blood pressure >95 mmHg

2. History of chronic renal insufficiency (serum creatinine >2.5 mg/dL)

3. History of liver disease or abnormal LFTs at screening (>3x upper limit of normal
[ULN])

4. Any major surgical procedure occurring < 3 months prior to the screening visit

5. Cardiac insufficiency defined by the New York Heart Association classification as
functional Class III or Class IV

6. History of a non-skin malignancy within the previous 5 years

7. History of alcohol or drug abuse

8. Participation in an investigational drug study within 6 weeks prior to the screening
visit

9. Serious or unstable medical or psychological conditions that, in the opinion of the
Investigator, would compromise the patient's safety or successful participation in the
study.



Age minimum: 13 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Homozygous Familial Hypercholesterolemia

Intervention(s)

Drug: Lomitapide

Primary Outcome(s)

LDL-C [Time Frame: Up to 16 weeks of treatment comapred to Baseline]

Secondary Outcome(s)

Absolute Change From Baseline in Vitamin D [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Hepatic Fat Percent [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Aspartate Aminotransferase (AST) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Total Bilirubin [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Alanine Aminotransferase (ALT) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Docosahexaenoic Acid (DHA) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Ratio of Vitamin E to Total Lipids [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Vitamin A [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Alpha Linoleic Acid (ALA) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Carbon Monoxide Lung Diffusing Capacity (DLCO)(a Pulmonary Function Test) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Eicosapentaenoic Acid (EPA) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Linoleic Acid (LA) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Forced Expiratory Volume During 1 Second (FEV1) [Time Frame: Baseline and 16 weeks of treatment]

Absolute Change From Baseline in Vitamin E [Time Frame: Baseline and 16 weeks of treatment]

Secondary ID(s)

UP1001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Doris Duke Charitable Foundation

University of Pennsylvania

Results

• Results available: Yes
• Date Posted: 22/02/2013
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01556906