Clinical trial • Phase II/III • Cardiology

ENLICITIDE CHLORIDE for Heterozygous familial hypercholesterolemia

Phase II/III trial of ENLICITIDE CHLORIDE for Heterozygous familial hypercholesterolemia.

Overview

Trial Therapeutic Area
Cardiology
Trial Disease
Heterozygous familial hypercholesterolemia
Trial Stage
Phase II/III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
18-09-2025
First CTIS Authorization Date
26-01-2026

Trial design

Randomised, placebo to mk-0616 (placebo to mk-0616 is the comparator arm). no dose or schedule for the placebo comparator is specified in the available ctis data.-controlled, adaptive Phase II/III trial in Belgium, Czechia, Finland and others.

Randomised
Yes
Comparator
Placebo to MK-0616 (Placebo to MK-0616 is the comparator arm). No dose or schedule for the placebo comparator is specified in the available CTIS data.
Adaptive
True - The study title and description denote an "Operationally Seamless Phase 2/3" design integrating Part A and Part B; no further adaptive rules (dose-escalation algorithm, interim analysis or stopping rules) are specified in the CTIS record.
Target Sample Size
149
Trial Duration For Participant
180

Eligibility

Recruits 149 paediatric patients.

Vulnerable Population
The trial enrolls pediatric participants (ages 6 to <18 years). Informed consent/assent materials are provided: parent/guardian consent forms and age‑appropriate assent forms are available (documents listed for age bands such as 6-9, 10-14, 12-13, 12-16, 14-17, 15-17, etc.). There are also forms for participants turning 18 and optional genetic consent forms. Consent and assent procedures therefore require parent/legally acceptable representative consent plus child assent appropriate to the participant's age; separate GDPR/data privacy information documents are provided.

Inclusion criteria

  • {"criterion_text":"- 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.\n- 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.\n- 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.\n- 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.\n- For Part A and Part B participants: Can take study medication by mouth and can swallow the study intervention.\n- 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

  • {"criterion_text":"- 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.\n- For Part A and Part B participants: Has a history of nephrotic syndrome.\n- For Part A and Part B participants: Has any clinically significant malabsorption condition.\n- For Part A and Part B participants: Has uncontrolled hypertension.\n- 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.\n- For Part B: Has ESRD on dialysis.\n- 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.\n- 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.\n- 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 endpoints

  • {"endpoint_text":"- Maximum Plasma Concentration (Cmax) of Enlicitide for Part A (Age Cohort 1)","definition_or_measurement_approach":"Plasma pharmacokinetic measurement: maximum observed plasma concentration (Cmax) of enlicitide in Part A Age Cohort 1."}
  • {"endpoint_text":"- Maximum Plasma Concentration (Cmax) of Enlicitide for Part A (Age Cohort 2)","definition_or_measurement_approach":"Plasma pharmacokinetic measurement: maximum observed plasma concentration (Cmax) of enlicitide in Part A Age Cohort 2."}
  • {"endpoint_text":"- Area Under the Concentration-time Curve From 0 to 24 (AUC0-24) Hours of Enlicitide for Part A (Age Cohort 1)","definition_or_measurement_approach":"Plasma pharmacokinetic measurement: area under the plasma concentration-time curve from 0 to 24 hours (AUC0-24) for enlicitide in Part A Age Cohort 1."}
  • {"endpoint_text":"- Area Under the Concentration-time Curve From 0 to 24 (AUC0-24) Hours of Enlicitide for Part A (Age Cohort 2)","definition_or_measurement_approach":"Plasma pharmacokinetic measurement: area under the plasma concentration-time curve from 0 to 24 hours (AUC0-24) for enlicitide in Part A Age Cohort 2."}
  • {"endpoint_text":"- Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 in Part B Participants","definition_or_measurement_approach":"Efficacy endpoint: percent change from baseline in LDL-C measured at Week 24 in Part B participants (comparing enlicitide with placebo)."}
  • {"endpoint_text":"- Percent Change From Baseline in LDL-C at Week 24 in Part B Age Cohort 1 Participants","definition_or_measurement_approach":"Efficacy endpoint: percent change from baseline in LDL-C measured at Week 24 in Part B Age Cohort 1 participants."}
  • {"endpoint_text":"- Number of Participants With One or More Adverse Events (AEs)","definition_or_measurement_approach":"Safety endpoint: count and proportion of participants experiencing one or more adverse events during the study period."}
  • {"endpoint_text":"- Number of Participants Who Discontinue Study Treatment Due to an AE","definition_or_measurement_approach":"Safety endpoint: count and proportion of participants who discontinue study treatment because of an adverse event."}

Secondary endpoints

  • {"endpoint_text":"- Percentage Change From Baseline in Apolipoprotein B (ApoB) at Week 24 in Part B Participants","definition_or_measurement_approach":"Percent change from baseline in ApoB measured at Week 24 in Part B participants."}
  • {"endpoint_text":"- Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 in Part B Participants","definition_or_measurement_approach":"Percent change from baseline in non-HDL-C measured at Week 24 in Part B participants."}
  • {"endpoint_text":"- Percentage Change From Baseline in Lipoprotein (a) [Lp (a)] at Week 24 in Part B Participants","definition_or_measurement_approach":"Percent change from baseline in Lp(a) measured at Week 24 in Part B participants."}
  • {"endpoint_text":"- Proportion of Participants With LDL-C <130 mg/dL (3.37 mmol/L) at Week 24 in Part B Participants","definition_or_measurement_approach":"Proportion of Part B participants with LDL-C <130 mg/dL at Week 24."}
  • {"endpoint_text":"- Proportion of Participants LDL-C ≥50% Reduction From Baseline at Week 24 in Part B Participants","definition_or_measurement_approach":"Proportion of Part B participants achieving ≥50% reduction from baseline in LDL-C at Week 24."}
  • {"endpoint_text":"- Proportion of Participants With LDL-C <100 mg/dL (2.59 mmol/L) at Week 24 in Part B Participants","definition_or_measurement_approach":"Proportion of Part B participants with LDL-C <100 mg/dL at Week 24."}
  • {"endpoint_text":"- Change in Ultrasound Determined Mean cIMT at Week 24 in Part B Participants","definition_or_measurement_approach":"Change from baseline in mean carotid intima-media thickness (cIMT) determined by ultrasound at Week 24 in Part B participants."}

Recruitment

Planned Sample Size
149
Recruitment Window Months
131
Consent Approach
Parental/guardian informed consent is required for minors, with age-appropriate assent documents provided for multiple age bands (examples in the dossier: assent forms for 6-9 yr, 6-11 yr, 6-12 yr, 10-14 yr, 10-17 yr, 12-13 yr, 12-16 yr, 12-17 yr, 14-17 yr, 15-17 yr). Separate consent forms are available for parents/guardians and for participants who reach age of majority (participant turn 18 forms). Optional consent modules (e.g., optional genetic testing consent, optional extension/OLE consent, pregnancy follow-up forms) and GDPR/data privacy information are provided. Documents are available in multiple country/language variants (English, French, Dutch, Spanish, German, Italian, Polish, Czech and local language versions as listed).

Methods

  • Site-based recruitment materials (posters) targeting potential participants and parents/caregivers — country-specific posters provided (e.g., Belgium: K2 Recruitment Doc Poster_BEL_EN/FR/NL).
  • Patient-facing printed materials (patient flyers, patient brochures, brochures) for distribution at sites to pediatric patients and parents/guardians — country-specific brochures/flyers listed (Belgium, Netherlands, Spain, France, Germany, Italy, Poland, Finland, Czechia, etc.).
  • Patient visit guides and site visit materials for recruitment and enrolment (documents listed under recruitment arrangements for several countries).

Geography

Total Number Of Sites
20
Total Number Of Participants
36

Belgium

Earliest CTIS Part Ii Submission Date
16-01-2026
Latest Decision Or Authorization Date
30-01-2026
Processing Time Days
14
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Universitair Ziekenhuis Antwerpen
Department Name
Pediatrics
Contact Person Name
Annick France
Contact Person Email
annick.france@uza.be
Site Name
UZ Leuven
Department Name
Pediatrics
Contact Person Name
Peter Witters
Contact Person Email
peter.witters@uzleuven.be

Czechia

Earliest CTIS Part Ii Submission Date
31-10-2025
Latest Decision Or Authorization Date
29-01-2026
Processing Time Days
90
Number Of Sites
3
Number Of Participants
6

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
Klinika pediatrie a dědičných poruch metabolismu (KPDPM) 1.LF UK a VFN
Contact Person Name
Pavel Ješina
Contact Person Email
pavel.jesina@vfn.cz
Site Name
Fakultni Nemocnice V Motole
Department Name
Ústav lékařské chemie a klinické biochemie 2. LF UK a FN Motol
Contact Person Name
Jana Čepová
Contact Person Email
jana.cepova@fnmotol.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Pediatrická klinika FN Brno a LF MU
Contact Person Name
Lenka Dostalová Kopečná

Finland

Earliest CTIS Part Ii Submission Date
12-12-2025
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
52
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
HUS-yhtymae
Department Name
HUS Lasten ja nuorten sairaudet Kliinisen tutkimuksen yksikkö
Contact Person Name
Risto Lapatto
Contact Person Email
risto.lapatto@hus.fi

France

Earliest CTIS Part Ii Submission Date
06-01-2026
Latest Decision Or Authorization Date
28-01-2026
Processing Time Days
22
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Trousseau Hospital
Department Name
gastroenterology and nutrition
Contact Person Name
Julie Lemale
Contact Person Email
julie.lemale@aphp.fr
Site Name
Hospices Civils De Lyon
Department Name
Paediatric hepatogoly, gastroenterology and nutrition
Contact Person Name
Noel Peretti
Contact Person Email
noel.peretti@chu-lyon.fr

Germany

Earliest CTIS Part Ii Submission Date
04-11-2025
Latest Decision Or Authorization Date
27-01-2026
Processing Time Days
84
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Medical Center - University Of Freiburg
Department Name
Kinder und Jugendklinik, Allgemeine Kinder- und Jugendmedizin
Contact Person Name
Clemens Kamrath
Site Name
Universitaetsklinikum Frankfurt AöR
Department Name
Klinik für Kinder- und Jugendmedizin
Contact Person Name
Ulrich Paetow
Site Name
Universitaetsklinikum Magdeburg AöR
Department Name
Kinderklinik, Bereich Endokrinologie und Diabetologie
Contact Person Name
Susann Empting
Contact Person Email
susann.empting@med.ovgu.de

Italy

Earliest CTIS Part Ii Submission Date
15-01-2026
Latest Decision Or Authorization Date
26-01-2026
Processing Time Days
11
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
Rare Deseases and Medical Genetics
Contact Person Name
Paola Sabrina Buonuomo
Contact Person Email
psabrina.buonuomo@opbg.net
Site Name
Azienda Unita Sanitaria Locale Di Piacenza
Department Name
UOC Pediatria e Neonatologia
Contact Person Name
Giacomo Biasucci
Contact Person Email
g.biasucci@ausl.pc.it

Netherlands

Earliest CTIS Part Ii Submission Date
15-12-2025
Latest Decision Or Authorization Date
26-01-2026
Processing Time Days
42
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Amsterdam UMC Stichting
Department Name
Vasular medicine clinical trial unit
Contact Person Name
Willemijn Corpeleijn
Contact Person Email
n.sons@amsterdamumc.nl

Norway

Earliest CTIS Part Ii Submission Date
10-12-2025
Latest Decision Or Authorization Date
30-01-2026
Processing Time Days
51
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Oslo University Hospital HF
Department Name
Lipidklinikken, Aker
Contact Person Name
Emil Asprusten
Contact Person Email
b22400@ous-hf.no

Poland

Earliest CTIS Part Ii Submission Date
19-12-2025
Latest Decision Or Authorization Date
30-01-2026
Processing Time Days
42
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Pediatrii, Diabetologii i Endokrynologii
Contact Person Name
Matylda Hennig
Site Name
Institute Of Polish Mother's Health Center
Department Name
Poradnia Wrodzonych Wad Metabolicznych u Dzieci Regionalne Centrum Chorób Rzadkich
Contact Person Name
Maciej Banach
Contact Person Email
bozena.sosnowska@umed.lodz.pl

Sponsor

Primary sponsor

Full Name
Merck Sharp & Dohme LLC
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Signant Health Global LLC
Responsibilities
3
Name
Eresearchtechnology Inc.
Responsibilities
Imaging
Name
Parexel International Corp.
Responsibilities
EUB services (call center and medical services)
Name
Labcorp Central Laboratory Services SARL
Responsibilities
Central laboratory services (code 4)
Name
Pharma Medica Research Inc.
Responsibilities
Code 4

Third parties

  • {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Imaging","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Stanford Quantitative Sciences Unit","duties_or_roles":"External Data Monitoring Committee","organisation_type":"Industry"}
  • {"country":"United States","full_name":"Parexel International Corp.","duties_or_roles":"EUB services (call center and medical services)","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
  • {"country":"Canada","full_name":"Pharma Medica Research Inc.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
MK-0616
Active Substance
ENLICITIDE CHLORIDE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Maximum Dose
20 mg (max daily dose amount 20 mg reported)
Investigational Product Name
Placebo to MK-0616
Modality
Other

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