A Study of MK-0616 (Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017)
Study Details
Study Description
Brief Summary
The goal of this study is to evaluate the efficacy, safety, and tolerability of MK-0616 in adult participants with heterozygous familial hypercholesterolemia. The primary hypothesis is that MK-0616 is superior to placebo on mean percent change from baseline in low-density lipoprotein cholesterol (LDL-C) at Week 24.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: MK-0616 Participants will receive 20 mg of MK-0616 orally once daily (QD) for up to 52 weeks. |
Drug: MK-0616
Oral tablet
|
| Placebo Comparator: Placebo Participants will receive MK-0616-matching placebo orally QD for up to 52 weeks. |
Drug: Placebo
Placebo
|
Outcome Measures
Primary Outcome Measures
- Mean percent change from baseline in low-density lipoprotein cholesterol (LDL-C) at Week 24 [Baseline and Week 24]
Blood samples will be collected at baseline and at Week 24 to assess mean percent change in LDL-C.
- Number of participants with one or more adverse events (AEs) [Up to ~60 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 drug due to an AE [Up to ~52 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 Outcome Measures
- Mean percent change from baseline in LDL-C at Week 52 [Baseline and Week 52]
Blood samples will be collected at baseline and at Week 52 to assess mean percent change in LDL-C.
- Mean percent change from baseline in non-high-density lipoprotein cholesterol (HDL-C) at Week 24 [Baseline and Week 24]
Blood samples will be collected at baseline and at Week 24 to assess mean percent change in non-HDL-C.
- Mean percent change from baseline in apolipoprotein B (ApoB) at Week 24 [Baseline and Week 24]
Blood samples will be collected at baseline and at Week 24 to assess mean percent change in ApoB.
- Percent change from baseline in lipoprotein(a) (Lp[a]) at Week 24 [Baseline and Week 24]
Blood samples will be collected at baseline and at Week 24 to assess percent change in Lp(a).
- Percentage of participants with LDL-C <70 mg/dL and ≥50% reduction from baseline at Week 24 [Baseline and Week 24]
Blood samples will be collected at baseline and at Week 24 to assess the percentage of participants who have LDL-C <70 mg/dL and ≥50% reduction from baseline.
- Percentage of participants with LDL-C <55 mg/dL and ≥50% reduction from baseline at Week 24 [Baseline and Week 24]
Blood samples will be collected at baseline and at Week 24 to assess the percentage of participants who have LDL-C <55 mg/dL and ≥50% reduction from baseline.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Has possible or definite diagnosis of heterozygous familial hypercholesterolemia (HeFH) based on a locally accepted diagnostic algorithm
-
Has an LDL-C ≥55 mg/dL or ≥70 mg/dL depending on medical history
-
Is treated with a moderate- or high-intensity statin medication
-
Is on a stable dose of all background lipid-lowering therapies (LLTs) with no planned medication change
Exclusion Criteria:
-
Has a history of homozygous familial hypercholesterolemia (FH) based on genetic or clinical criteria, compound heterozygous FH, or double heterozygous FH
-
Has a history of heart failure or heart failure hospitalization within 3 months before first study visit
-
Is undergoing or previously underwent an LDL-C apheresis program within 3 months before first study visit or plans to initiate an LDL-C apheresis program
-
Was previously treated/is being treated with certain other cholesterol lowering medications, including protein convertase subtilisin/kexin type 9 (PCSK9) inhibitors
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Merck Sharp & Dohme LLC
Investigators
- Study Director: Medical Director, Merck Sharp & Dohme LLC
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- 0616-017
- MK-0616-017
- 2022-502782-14
- U1111-1285-4257