Ignite Creation Date:
2025-12-25 @ 2:07 AM
Ignite Modification Date:
2025-12-26 @ 3:19 AM
Study NCT ID:
NCT05694260
Status:
COMPLETED
Last Update Posted:
2025-08-08
First Post:
2023-01-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006937', 'term': 'Hypercholesterolemia'}], 'ancestors': [{'id': 'D006949', 'term': 'Hyperlipidemias'}, {'id': 'D050171', 'term': 'Dyslipidemias'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C581236', 'term': '8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 31}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-01-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2025-06-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-07', 'studyFirstSubmitDate': '2023-01-12', 'studyFirstSubmitQcDate': '2023-01-12', 'lastUpdatePostDateStruct': {'date': '2025-08-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-01-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-06-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Plasma trough concentration of ETC-1002', 'timeFrame': '8 weeks of steady-state dosing'}, {'measure': 'Area under the plasma concentration-time curve (AUC,ss) of ETC-1002', 'timeFrame': '8 weeks of steady-state dosing'}, {'measure': 'Average plasma concentration (Cavg,ss) of ETC-1002', 'timeFrame': '8 weeks of steady-state dosing'}, {'measure': 'Maximum plasma concentration (Cmax,ss) of ETC-1002', 'timeFrame': '8 weeks of steady-state dosing'}], 'secondaryOutcomes': [{'measure': 'Plasma trough concentration of ESP15228', 'timeFrame': '8 weeks of steady-state dosing'}, {'measure': 'Plasma concentration at 4 hours (C4hr) of ETC-1002', 'timeFrame': 'Day 1'}, {'measure': 'C4hr of ESP15228', 'timeFrame': 'Day 1'}, {'measure': 'Exposure/LDL-C response relationship', 'timeFrame': '8 weeks of steady-state dosing', 'description': 'ETC-1002 dose and exposure/LDL-C-lowering response relationship'}, {'measure': 'Percent change from Baseline in low-density lipoprotein cholesterol (LDL-C)', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Absolute change from Baseline in LDL-C', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Percent change from Baseline in non-high-density lipoprotein cholesterol (non-HDL-C)', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Absolute change from Baseline in non-HDL-C', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Percent change from Baseline in total cholesterol (TC)', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Absolute change from Baseline in TC', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Percent change from Baseline in high-sensitivity C-reactive protein (hsCRP)', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Absolute change from Baseline in hsCRP', 'timeFrame': 'Baseline and at Weeks 8 and 16'}, {'measure': 'Acceptability of taste using a dosing acceptability questionnaire', 'timeFrame': 'Up to Week 16'}, {'measure': 'Acceptability of ease of swallowing using a dosing acceptability questionnaire', 'timeFrame': 'Up to Week 16'}, {'measure': 'Number of participants reporting Serious adverse events (SAEs) and non-SAEs', 'timeFrame': 'Up to Week 16'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Pediatric', 'Heterozygous familial hypercholesterolemia', 'Low-density lipoprotein cholesterol (LDL-cholesterol)', 'Bempedoic acid', 'ETC-1002', 'Adenosine triphosphate citrate lyase'], 'conditions': ['Hypercholesterolemia']}, 'descriptionModule': {'briefSummary': 'Multiple-dose study to measure pharmacokinetics, pharmacodynamics and safety of bempedoic acid in pediatric participants 6 to 17 years of age with HeFH.', 'detailedDescription': 'Dose-selection based on body weight will be determined for use in pediatric clinical development.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Participant's parent(s)/guardian(s) must be willing to provide written informed consent and the participant must provide informed assent before any study-specific procedures are performed;\n* Participant must be aged 6-17 years old and willing to swallow tablets;\n* Participant must weigh at least 16 kilograms (kg);\n* Participant must have a diagnosis of HeFH prior to receiving the first dose of study medication at Treatment Visit T1 per Make Early Diagnosis to Prevent Early Deaths project (MEDPED) criteria by meeting at least one of the following clinical criteria:\n\n 1. Documented diagnosis of HeFH determined by positive genetic testing; or\n 2. Documented LDL-C or TC meeting one or more of the following criteria:\n\n i. LDL-C \\>200 milligrams per deciliter (mg/dL) (5.2 millimole per liter \\[mmol/L\\]) or TC \\>270 mg/dL (7.0 mmol/L), with no first- second- or third-degree relative with documented FH diagnosis (general population); or ii. LDL-C \\>155 mg/dL (4.0 mmol/L) or TC \\>220 mg/dL (5.7 mmol/L), and also having a first-degree relative with documented familial hypercholesterolemia (FH) diagnosis; or iii. LDL-C \\>165 mg/dL (4.3 mmol/L) or TC \\>230 mg/dL (5.9 mmol/L), and also having a second-degree relative with documented FH diagnosis; or iv. LDL-C \\>170 mg/dL (4.4 mmol/L) or TC \\>240 mg/dL (6.2 mmol/L), and also having a third-degree relative with documented FH diagnosis\n* Current treatment with approved stable lipid-modifying therapy (LMT), including an optimal dose of statin with or without other LMT(s), at stable dose for at least 4 weeks prior to Treatment Visit T1 (6 weeks for fibrates; however, gemfibrozil is not allowed in participants taking a statin as per coadministration instructions defined in the statin label). Participants must remain on that stable dose throughout the duration of the trial. Optimal dose of statin will be determined by the investigator using their medical judgment and available sources, including the participant's self-reported history of LMT. A participant's optimal dose of statin is defined as meeting one of the following criteria:\n\n 1. the highest approved dose of statin prescribed for the age of the participant based on regional practice or local guidelines; or\n 2. less than the highest approved dose of statin, including no statin, prescribed for the age of the participant based on regional practice or local guidelines (including no statin) if: i. the participant has previously taken 2 or more statin therapies at any dose and not able to tolerate or unresponsive due to their mutations (null); or ii. the participant has previously taken 1 or more statin therapies at any dose and is unwilling to attempt another statin at any dose or advised by a physician to not attempt another statin at any dose.\n 3. Participant/parent and investigator attestation to the participant's unwillingness to attempt and/or physician advice to not attempt additional statin therapy will be recorded.\n\nExclusion Criteria:\n\n* Participant has a diagnosis of homozygous familial hypercholesterolemia (HoFH) or compound HeFH;\n* Participant has a fasting triglyceride (TG) level ≥400 mg/dL (4.5 mmol/L);\n* Participant has uncontrolled hypothyroidism, including a value for thyroid-stimulating hormone (TSH) \\< lower limit of normal (LLN) or \\>1.5 × the upper limit of normal (ULN);\n* Participant has liver disease or dysfunction, including:\n\n 1. positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis C virus antibodies (HCV-AB), or\n 2. serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value ≥2 × ULN and/or serum total bilirubin (TB) value ≥2 × ULN. If the serum TB value is ≥1.2 × ULN, a reflex indirect (unconjugated) bilirubin will be obtained and, if consistent with Gilbert's disease or if the participant has a history of Gilbert's disease, the participant may be enrolled in the study.\n* Participant has renal dysfunction or glomerulonephritis, including an estimated glomerular filtration rate (eGFR) \\<75 milliliters/minute/1.73 square meter (mL/min/1.73 m\\^2).\n\nOther protocol defined inclusion and exclusion criteria."}, 'identificationModule': {'nctId': 'NCT05694260', 'acronym': 'CLEAR Path 1', 'briefTitle': 'A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Esperion Therapeutics, Inc.'}, 'officialTitle': 'An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic Acid in Pediatric Patients (6 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia', 'orgStudyIdInfo': {'id': '1002-041'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'Participants at 16 to \\<30 kilograms (kg) body weight at screening receiving once daily 60 milligrams (mg) bempedoic acid for 8 weeks followed by 90 mg bempedoic acid for 8 weeks.', 'interventionNames': ['Drug: Bempedoic acid']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'Participants at 30 to 60 kg body weight at screening receiving once daily120 mg bempedoic acid for 8 weeks followed by 150 mg bempedoic acid for 8 weeks.', 'interventionNames': ['Drug: Bempedoic acid']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': 'Participants at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks.', 'interventionNames': ['Drug: Bempedoic acid']}], 'interventions': [{'name': 'Bempedoic acid', 'type': 'DRUG', 'otherNames': ['ETC-1002'], 'description': 'Once daily oral dosing with oral tablets.', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90048', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Cedars-Sinai Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '91790', 'city': 'West Covina', 'state': 'California', 'country': 'United States', 'facility': 'Providere Research Inc', 'geoPoint': {'lat': 34.06862, 'lon': -117.93895}}, {'zip': '33434', 'city': 'Boca Raton', 'state': 'Florida', 'country': 'United States', 'facility': 'Excel Medical Clinical Trials, LLC', 'geoPoint': {'lat': 26.35869, 'lon': -80.0831}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '27157', 'city': 'Winston-Salem', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Wake Forest University Health Sciences', 'geoPoint': {'lat': 36.09986, 'lon': -80.24422}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '17601', 'city': 'Lancaster', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Cardiology Care for Children', 'geoPoint': {'lat': 40.03788, 'lon': -76.30551}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': "University of Utah and Primary Children's Hospital", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': 'T6G 2B7', 'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': "University of Alberta Hospital - Stollery Children's Hospital", 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}, {'zip': 'L8N 3Z5', 'city': 'Hamilton', 'state': 'Ontario', 'country': 'Canada', 'facility': 'McMaster University Medical Center', 'geoPoint': {'lat': 43.25011, 'lon': -79.84963}}, {'zip': 'G7H 5H6', 'city': 'Chicoutimi', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Ecogene-21', 'geoPoint': {'lat': 48.41963, 'lon': -71.06369}}, {'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Rigshospitalet', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'city': 'Viborg', 'country': 'Denmark', 'facility': 'Viborg Regional Hospital', 'geoPoint': {'lat': 56.45319, 'lon': 9.40201}}, {'city': 'Frankfurt am Main', 'country': 'Germany', 'facility': 'Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet', 'geoPoint': {'lat': 50.11552, 'lon': 8.68417}}, {'city': 'Hanover', 'country': 'Germany', 'facility': 'Kinder- und Jugendkrankenhaus AUF DER BULT', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'zip': '1105 AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Amsterdam UMC - Locatie AMC', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': '3015 G', 'city': 'Rotterdam', 'country': 'Netherlands', 'facility': 'Erasmus MC', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'zip': '15001', 'city': 'A Coruña', 'state': 'Galicia', 'country': 'Spain', 'facility': 'Hospital Abente y Lago', 'geoPoint': {'lat': 43.37135, 'lon': -8.396}}, {'zip': '8208', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Corporacio Sanitaria Parc Tauli - Hospital de Sabadell', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '8950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Deu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '11407', 'city': 'Cadiz', 'country': 'Spain', 'facility': 'Hospital Universitario de Jerez de la Frontera', 'geoPoint': {'lat': 36.52672, 'lon': -6.2891}}, {'zip': '14004', 'city': 'Córdoba', 'country': 'Spain', 'facility': 'Hospital Universitario Reina Sofia', 'geoPoint': {'lat': 37.89155, 'lon': -4.77275}}, {'zip': '28034', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario Ramon y Cajal', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario 12 de Octubre', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': "The Investigator must ensure that the participant's confidentiality is maintained. The names and identities of all research participants will be kept in strict confidence and will not appear on eCRFs or other records that are provided to or retained by the Sponsor (or designee). If a participant's name appears on any document, it must be redacted and replaced with the participant identifier before a copy of the document is supplied to the Sponsor (or designee). The ICF must include appropriate statements explaining that participant data will be confidential and the actions that will be taken to ensure participant confidentiality.\n\nAny other confidentiality requirements specified by the site, IRB or IEC, or national or local regulations will be adhered to and detailed appropriately in the ICF."}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Esperion Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}