Heterozygous Familial Hypercholesterolemia (HeFH)
Conditions
Brief summary
This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density lipoprotein cholesterol (LDL-C) in the blood. The goals of this study are to learn about the safety of enlicitide and if children tolerate it, what happens to enlicitide in a child's body over time, and if enlicitide works to lower cholesterol levels in children more than a placebo.
Interventions
Enlicitide decanoate taken by mouth
DRUGPlacebo
Placebo tablet matched to enlicitide decanoate taken by mouth
Sponsors
Merck Sharp & Dohme LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Part A and the extension period are open-label. Part B is double-blinded.
Intervention model description
There are two parts of the study. Part A is a non randomized single treatment group. Part B is randomized parallel treatment groups. Participants who complete either part are then eligible to enroll in an open label extension (single group).
Eligibility
Sex/Gender
ALL
Age
6 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
Inclusion criteria include, but are not limited to: * Has possible or definite diagnosis of HeFH based on a locally accepted diagnostic algorithm or diagnosis by genetic testing results * Has a fasted LDL-C value (evaluated by the central laboratory) that is ≥130 mg/dL * Is receiving either: * An optimized daily dose of statin (± nonstatin LLT) * A nonstatin LLT with documented intolerance to at least 2 different statins, or documented intolerance to 1 statin plus refusal of statin therapy by the participant or legally acceptable representative * Is on a stable dose of all background LLTs for at least 30 days prior to screening, with no medication or dose changes planned during participation in Part A or Part B
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Maximum Plasma Concentration (Cmax) of Enlicitide | At designated timepoints (up to 24 hours postdose on day 14) | Blood samples will be collected to determine the Cmax of enlicitide. |
| Part A: Area Under the Concentration-Time Curve from 0 to 24 Hours (AUC0-24) of Enlicitide | At designated timepoints (up to 24 hours postdose on day 14) | Blood samples will be collected to determine the AUC0-24 of enlicitide. |
| Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 24 | Blood samples will be collected to determine the percent change from baseline in LDL-C. |
| Number of Participants Who Experience an Adverse Event (AE) | Up to approximately 188 weeks | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. |
| Number of Participants Who Discontinue Study Treatment Due to an AE | Up to approximately 180 weeks | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part B: Percent Change from Baseline in Apolipoprotein B (ApoB) | Baseline and week 24 | Blood samples will be collected to determine the percent change from baseline in apolipoprotein B. |
| Part B: Percent Change from Baseline in Non-High-Density Lipoprotein Cholesterol (non-HDL-C) | Baseline and week 24 | Blood samples will be collected to determine the percent change from baseline in non-HDL-C. |
| Part B: Percent Change from Baseline in Lipoprotein (a) (Lp(a)) | Baseline and week 24 | Blood samples will be collected to determine the percent change from baseline in Lp(a). |
| Part B: Percentage of Participants With LDL-C <130 mg/dL at Week 24 | Week 24 | The percentage of participants with LDL-C \<130 mg/dL at week 24 will be reported. |
| Part B: Percentage of Participants With ≥50% LDL-C Reduction from Baseline at Week 24 | Baseline and week 24 | The percentage of participants with ≥50% LDL-C reduction from baseline at week 24 will be reported. |
| Part B: Percentage of Participants With LDL-C <100 mg/dL at Week 24 | Week 24 | The percentage of participants with LDL-C \<100 mg/dL at week 24 will be reported. |
| Change in Carotid Intima-media Thickness (cIMT) | Baseline and week 24 | Ultrasound measurements will be performed to determine the change from baseline in cIMT. |
Countries
Australia, Belgium, Brazil, Chile, China, Colombia, Czechia, Finland, France, Germany, Mexico, Netherlands, New Zealand, Poland, Singapore, Spain, United Kingdom, United States
Contacts
CONTACTToll Free Number
STUDY_DIRECTORMedical Director
Merck Sharp & Dohme LLC
Outcome results
None listed