A clinical study of enlicitide in children with high cholesterol (MK-0616-029)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Merck Sharp & Dohme LLC

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

12 Feb 2026 → ongoing

Decision date (initial)

2026-01-30

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Merck Sharp & Dohme LLC

External identifiers

EU CT number

2024-519068-42-00

WHO UTN

U1111-1314-5796

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Pharmacodynamic, Therapy, Pharmacokinetic

1. Part A (Age Cohort 1 and Age Cohort 2 separately): To evaluate the pharmacokinetics of enlicitide.

2. Part B (Age Cohort 1 and Age Cohort 2 combined): To evaluate the efficacy of enlicitide compared with placebo on percent change from baseline in LDL-C at Week 24.

3. Part B Age Cohort 1: To evaluate the efficacy of enlicitide compared with placebo on percent change from baseline in LDL-C at Week 24.

4. Part A, Part B, and OLE: To evaluate the safety and tolerability of enlicitide.

Secondary objectives 3

1. Part B: To evaluate the efficacy of enlicitide compared with placebo on percent change from baseline in ApoB, non-HDLC, and Lp(a) at Week 24.
2. Part B: To evaluate the efficacy of enlicitide compared with placebo on the proportion of participants with LDL-C meeting the following goals at Week 24: • <130 mg/dL (3.37 mmol/L). • ≥50% reduction from baseline. • <100 mg/dL (2.59 mmol/L).
3. Part B: To evaluate the efficacy of enlicitide compared with placebo on change in ultrasound determined mean carotid intima-media thickness (cIMT) at Week 24.

Conditions and MedDRA coding

​Heterozygous Familial Hypercholesterolemia​ in pediatric patients

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003453-PIP01-23

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. For Part A and Part B participants: Has possible or definite diagnosis of heterozygous familial hypercholesterolemia (HeFH) based on a locally accepted diagnostic algorithm or diagnosis by genetic testing results before screening.
2. For Part A and Part B participants: Is receiving optimized treatment per local guidelines, standard of care, and investigator judgment with an optimized daily dose of statin [(± non-statin lipid-lowering treatment (LLT)] OR non-statin LLT with either a documented intolerance to at least 2 different statins, or refusal of statin therapy by the participant or legally acceptable representative and with written attestation.
3. For Part A and Part B participants: Is on a stable dose of all background LLTs (including statin and non-statin agents) before screening and through allocation/randomization with no medication or dose changes planned.
4. For Part A and Part B participants: Are 12 to <18 years of age for Age Cohort 1 and 6 to <12 years of age for Age Cohort 2.
5. For Part A and Part B participants: Can take study medication by mouth and can swallow the study intervention.
6. For open-label extension (OLE) period participants: Is a Part A participant who received at least 1 dose of study intervention and completed Visit 5 (Day 14) OR is a Part B participant who received at least 1 dose of study intervention and completed Visit 7 (Week 24).

Exclusion criteria 9

1. For Part A and Part B participants: Has a history of homozygous familial hypercholesterolemia (FH) based on genetic or clinical criteria or history of known compound heterozygous FH, or double heterozygous FH.
2. For Part A and Part B participants: Has a history of nephrotic syndrome.
3. For Part A and Part B participants: Has any clinically significant malabsorption condition.
4. For Part A and Part B participants: Has uncontrolled hypertension.
5. For Part A: Has severe chronic kidney disease defined as estimated glomerular filtration rate (eGFR) <30 ml/min/1.73 m2 or end stage renal disease (ESRD) on dialysis.
6. For Part B: Has ESRD on dialysis.
7. For Part A, Part B and OLE Period: Is undergoing or previously underwent an LDL-C apheresis program within 3 months before visit 1 or plans to initiate an LDL-C apheresis program.
8. For Part A, Part B and OLE Period: Is currently participating in or has previously participated in an interventional clinical study within 3 months before visit 1.
9. For Part A, Part B and OLE Period: If participant has enrolled in Part A they cannot enroll in Part B and vice versa.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 8

1. Maximum Plasma Concentration (Cmax) of Enlicitide for Part A (Age Cohort 1)
2. Maximum Plasma Concentration (Cmax) of Enlicitide for Part A (Age Cohort 2)
3. Area Under the Concentration-time Curve From 0 to 24 (AUC0-24) Hours of Enlicitide for Part A (Age Cohort 1)
4. Area Under the Concentration-time Curve From 0 to 24 (AUC0-24) Hours of Enlicitide for Part A (Age Cohort 2)
5. Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 in Part B Participants
6. Percent Change From Baseline in LDL-C at Week 24 in Part B Age Cohort 1 Participants
7. Number of Participants With One or More Adverse Events (AEs)
8. Number of Participants Who Discontinue Study Treatment Due to an AE

Secondary endpoints 7

1. Percentage Change From Baseline in Apolipoprotein B (ApoB) at Week 24 in Part B Participants
2. Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 in Part B Participants
3. Percentage Change From Baseline in Lipoprotein (a) [Lp (a)] at Week 24 in Part B Participants
4. Proportion of Participants With LDL-C <130 mg/dL (3.37 mmol/L) at Week 24 in Part B Participants
5. Proportion of Participants LDL-C ≥50% Reduction From Baseline at Week 24 in Part B Participants
6. Proportion of Participants With LDL-C <100 mg/dL (2.59 mmol/L) at Week 24 in Part B Participants
7. Change in Ultrasound Determined Mean cIMT at Week 24 in Part B Participants

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merck Sharp & Dohme LLC

Sponsor organisation

Merck Sharp & Dohme LLC

Address

126 East Lincoln Avenue, P. O. Box 2000 P. O. Box 2000

City

Rahway

Postcode

07065-4607

Country

United States

Scientific contact point

Organisation

Merck Sharp & Dohme LLC

Contact name

​Laura Gellis​

Public contact point

Organisation

Merck Sharp & Dohme LLC

Contact name

​Laura Gellis​

Third parties 6

Locations

10 EU/EEA countries · 20 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 170 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications