- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03038022
Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
January 23, 2018 updated by: Madrigal Pharmaceuticals, Inc.
A Phase 2, Multi-Center, Double-Blind, Randomized, Placebo Controlled Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia
The primary objective of this study is to determine the effect of once-daily oral MGL-3196 on the percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in patients with Heterozygous Familial Hypercholesterolemia (HeFH).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
116
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Aalborg, Denmark
- Madrigal Research Site
-
Viborg, Denmark
- Madrigal Research Site
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Must be willing to participate in the study and provide written informed consent;
- Male and female adults ≥18 years of age;
- Female patients of child bearing potential with negative serum pregnancy (beta human chorionic gonadotropin) test who are not breastfeeding, do not plan to become pregnant during the study, and agree to use effective birth control (ie, condoms, diaphragm, non-hormonal intrauterine device [IUD], or sexual abstinence [only if this is in line with the patient's current lifestyle]) throughout the study and for at least 1 month after study completion; hormonal contraception (estrogens stable ≥3 months) and hormonal IUDs are permitted if used with a secondary birth control measure (eg, condoms); OR female patients of non-child bearing potential (ie, surgically [bilateral oophorectomy, hysterectomy, or tubal ligation] or naturally sterile [>12 consecutive months without menses]); male patients who have sexual intercourse with a female partner of child bearing potential from the first dose of study drug until 1 month after study completion must either be surgically sterile (confirmed by documented azoospermia >90 days after the procedure) OR agree to use a condom with spermicide. All male patients must agree not to donate sperm from the first dose of study drug until 1 month after study completion;
- Must have a diagnosis of HeFH by genetic testing or by having met the diagnostic criteria for definite familial hypercholesterolemia outlined by the Simon Broome Register Group or WHO/Dutch Lipid Network (score >8);
- Must have a fasting LDL-C ≥ 2.6 mmol/L (100 mg/dL); and
- Must be on a stable or maximally tolerated dose (≥ 4 weeks prior to screening) of an approved statin (rosuvastatin ≤ 20 mg daily, atorvastatin ≤ 80 mg daily), with or without ezetimibe.
Exclusion Criteria:
- Homozygous familial hypercholesterolemia
- Low-density lipoprotein (LDL) or plasma apheresis within 2 months prior to randomization;
- New York Heart Association class III or IV heart failure, or known left ventricular ejection fraction <30%;
- Uncontrolled cardiac arrhythmia, including confirmed QT interval corrected using Fridericia's formula (QTcF) >450 msec for males and >470 msec for females at the screening electrocardiogram (ECG) assessment;
- Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft, or stroke within 3 months prior to randomization;
- Type 1 diabetes, or newly diagnosed or uncontrolled type 2 diabetes (hemoglobin A1c [HbA1c] >8%);
- History of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to screening; Note: Significant alcohol consumption is defined as average of >20 g/day in female patients and >30 g/day in male patients;
- Thyroid replacement therapy;
- Evidence of chronic liver disease;
- Hepatitis B, as defined by the presence of hepatitis B surface antigen;
- Hepatitis C, as defined by the presence of hepatitis C virus (HCV) antibody (anti-HCV) and HCV ribonucleic acid (RNA). Patients with positive anti-HCV who test negative for HCV RNA at screening will be allowed to participate in the study;
- Serum alanine aminotransferase (ALT) >1.5 × ULN (one repeat allowed);
- Estimated glomerular filtration rate <60 mL/min;
- Creatine kinase >3 × ULN (one repeat allowed);
- History of biliary diversion;
- Positive for human immunodeficiency virus infection;
- History of malignant hypertension;
- Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at screening or randomization and confirmed at an unscheduled visit;
- Triglycerides >5.7 mmol/L (500 mg/dL) at screening and confirmed by repeat assessment;
- Active, serious medical disease with likely life expectancy <2 years;
- Active substance abuse, including inhaled or injection drugs within the year prior to screening;
- Participation in an investigational new drug trial within the 30 days prior to randomization; or
- Any other condition which, in the opinion of the Investigator, would impede compliance, hinder completion of the study, or compromise the well-being of the patient.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
Matching Placebo
|
Oral
|
Experimental: MGL-3196
Study Drug
|
Oral
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Mean percent change from baseline in low-density lipoprotein cholesterol (LDL-C)
Time Frame: 12 Weeks
|
12 Weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety and tolerability of MGL-3196 based on Adverse Events and Changes in Laboratory Values
Time Frame: 12 Weeks
|
12 Weeks
|
|
Mean percent change from baseline on selected lipid parameters
Time Frame: 12 Weeks
|
Non-high-density lipoprotein cholesterol (non-HDL-C),Apolipoprotein B (ApoB), Total cholesterol (TC)/high-density lipoprotein cholesterol (HDL-C) ratio, Triglycerides,Lipoprotein(a), Apolipoprotein A1 (ApoA1)/ApoB ratio, and Lipoprotein particle assessment
|
12 Weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Rebecca Taub, MD, Madrigal Pharmaceuticals, Inc.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 9, 2017
Primary Completion (Actual)
December 18, 2017
Study Completion (Actual)
January 15, 2018
Study Registration Dates
First Submitted
January 30, 2017
First Submitted That Met QC Criteria
January 30, 2017
First Posted (Estimate)
January 31, 2017
Study Record Updates
Last Update Posted (Actual)
January 25, 2018
Last Update Submitted That Met QC Criteria
January 23, 2018
Last Verified
January 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MGL-3196-06
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
Yes
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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