Phase
Condition
Familial Hypercholesterolemia
Hypercholesterolemia
Treatment
Bempedoic acid
Clinical Study ID
Ages 6-17 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Participant's parent(s)/guardian(s) must be willing to provide written informedconsent and the participant must provide informed assent before any study-specificprocedures are performed;
Participant must be aged 6-17 years old and willing to swallow tablets;
Participant must weigh at least 16 kilograms (kg);
Participant must have a diagnosis of HeFH prior to receiving the first dose of studymedication at Treatment Visit T1 per Make Early Diagnosis to Prevent Early Deathsproject (MEDPED) criteria by meeting at least one of the following clinicalcriteria:
Documented diagnosis of HeFH determined by positive genetic testing; or
Documented LDL-C or TC meeting one or more of the following criteria: i. LDL-C >200 milligrams per deciliter (mg/dL) (5.2 millimole per liter [mmol/L]) orTC >270 mg/dL (7.0 mmol/L), with no first- second- or third-degree relative withdocumented 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 withdocumented 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 relativewith 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
- Current treatment with approved stable lipid-modifying therapy (LMT), including anoptimal dose of statin with or without other LMT(s), at stable dose for at least 4weeks prior to Treatment Visit T1 (6 weeks for fibrates; however, gemfibrozil is notallowed in participants taking a statin as per coadministration instructions definedin the statin label). Participants must remain on that stable dose throughout theduration of the trial. Optimal dose of statin will be determined by the investigatorusing their medical judgment and available sources, including the participant'sself-reported history of LMT. A participant's optimal dose of statin is defined asmeeting one of the following criteria:
the highest approved dose of statin prescribed for the age of the participantbased on regional practice or local guidelines; or
less than the highest approved dose of statin, including no statin, prescribedfor the age of the participant based on regional practice or local guidelines (including no statin) if: i. the participant has previously taken 2 or morestatin therapies at any dose and not able to tolerate or unresponsive due totheir mutations (null); or ii. the participant has previously taken 1 or morestatin therapies at any dose and is unwilling to attempt another statin at anydose or advised by a physician to not attempt another statin at any dose.
Participant/parent and investigator attestation to the participant'sunwillingness to attempt and/or physician advice to not attempt additionalstatin therapy will be recorded.
Exclusion
Exclusion Criteria:
Participant has a diagnosis of homozygous familial hypercholesterolemia (HoFH) orcompound HeFH;
Participant has a fasting triglyceride (TG) level ≥400 mg/dL (4.5 mmol/L);
Participant has uncontrolled hypothyroidism, including a value forthyroid-stimulating hormone (TSH) < lower limit of normal (LLN) or >1.5 × the upperlimit of normal (ULN);
Participant has liver disease or dysfunction, including:
positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis Cvirus antibodies (HCV-AB), or
serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value ≥2 × ULN and/or serum total bilirubin (TB) value ≥2 × ULN. If the serum TBvalue is ≥1.2 × ULN, a reflex indirect (unconjugated) bilirubin will beobtained and, if consistent with Gilbert's disease or if the participant has ahistory of Gilbert's disease, the participant may be enrolled in the study.
- Participant has renal dysfunction or glomerulonephritis, including an estimatedglomerular filtration rate (eGFR) <75 milliliters/minute/1.73 square meter (mL/min/1.73 m^2).
Other protocol defined inclusion and exclusion criteria.
Study Design
Study Description
Connect with a study center
University of Alberta Hospital - Stollery Children's Hospital
Edmonton, Alberta T6G 2B7
CanadaActive - Recruiting
McMaster University Medical Center
Hamilton, Ontario L8N 3Z5
CanadaActive - Recruiting
The Hospital for Sick Children (SickKids)
Toronto, Ontario M5G 1X8
CanadaSite Not Available
Ecogene-21
Chicoutimi, Quebec G7H 5H6
CanadaActive - Recruiting
Rigshospitalet
Copenhagen, 2100
DenmarkActive - Recruiting
Viborg Regional Hospital
Viborg,
DenmarkActive - Recruiting
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
Frankfurt am Main,
GermanyActive - Recruiting
Kinder- und Jugendkrankenhaus AUF DER BULT
Hannover,
GermanyActive - Recruiting
Amsterdam UMC - Locatie AMC
Amsterdam, 1105 AZ
NetherlandsActive - Recruiting
Erasmus MC
Rotterdam, 3015 G
NetherlandsActive - Recruiting
Hospital Abente y Lago
La Coruña, Galicia 15001
SpainActive - Recruiting
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Barcelona, 8208
SpainActive - Recruiting
Hospital Sant Joan de Deu
Barcelona, 8950
SpainActive - Recruiting
Hospital Universitario de Jerez de la Frontera
Cadiz, 11407
SpainActive - Recruiting
Hospital Universitario Reina Sofia
Córdoba, 14004
SpainActive - Recruiting
Hospital Universitario 12 de Octubre
Madrid,
SpainActive - Recruiting
Hospital Universitario Ramon y Cajal
Madrid, 28034
SpainActive - Recruiting
Cedars-Sinai Medical Center
Los Angeles, California 90048
United StatesActive - Recruiting
Smidt Heart Institute at Cedars-Sinai
Los Angeles, California 90048
United StatesActive - Recruiting
Providere Research Inc
West Covina, California 91790
United StatesActive - Recruiting
Excel Medical Clinical Trials, LLC
Boca Raton, Florida 33434
United StatesActive - Recruiting
Continental Clinical Solutions
Towson, Maryland 21204
United StatesSite Not Available
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research.
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Wake Forest University Health Sciences
Winston-Salem, North Carolina 27157
United StatesActive - Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesActive - Recruiting
Cardiology Care for Children
Lancaster, Pennsylvania 17601
United StatesActive - Recruiting
University of Utah and Primary Children's Hospital
Salt Lake City, Utah 84113
United StatesActive - Recruiting
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.