A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
Purpose
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.
Condition
- Heterozygous Familial Hypercholesterolemia (HeFH)
Eligibility
- Eligible Ages
- Between 6 Years and 17 Years
- Eligible Sex
- All
- Accepts 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); or a nonstatin LLT with documented intolerance to at least 2 different statins or 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
Exclusion Criteria
Exclusion criteria include, but are not limited to: - Has a history of homozygous FH based on genetic or clinical criteria, or history of known compound heterozygous FH, or double heterozygous FH - Has a history of nephrotic syndrome - Has any clinically significant malabsorption condition based on principal investigator assessment - Was previously treated/is being treated with certain other cholesterol lowering medications, including proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors without adequate washout
Study Design
- Phase
- Phase 2/Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- 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).
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Masking Description
- Part A and the extension period are open-label. Part B is double-blinded.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Part A: Enlicitide Decanoate |
Participants receive enlicitide decanoate orally once daily (QD) at a dosage determined by age for up to 2 weeks. |
|
|
Experimental Part B: Enlicitide Decanoate |
Participants receive enlicitide decanoate QD at a dosage determined by age for up to 24 weeks. |
|
|
Placebo Comparator Part B: Placebo |
Participants receive placebo orally QD for up to 24 weeks. |
|
|
Experimental Open-Label Extension: Enlicitide Decanoate |
Participants who complete either Part A or Part B may enroll in this open-label extension arm. Participants in the extension arm receive enlicitide decanoate QD at a dosage determined by age for up to 3 years. |
|
Recruiting Locations
Children's National and nearby locations
Washington D.C. 4140963, District of Columbia 4138106 20010
Study Coordinator
202-476-5000
More Details
- NCT ID
- NCT07058077
- Status
- Recruiting
- Sponsor
- Merck Sharp & Dohme LLC