Ended Therapeutic exploratory (Phase II) Heterozygous familial hypercholesterolemia

A Phase 2 Clinical Study in Children With HeFH Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)

EU CTIS ID: 2024-515864-30-00

What this study is testing

To assess the pharmacokinetics (PK) of bempedoic acid (ETC-1002) in pediatric patients (6 to 17 years of age) with HeFH treated for 8 weeks.

  • Therapeutic exploratory (Phase II)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • The patient's parent(s)/guardian(s) must be willing to provide written informed consent and the patient must provide informed assent before any study-specific procedures are performed
  • The patient must be aged 6-17 years old and willing to swallow tablets
  • The patient must weigh at least 16 kg
  • The patient must have a diagnosis of HeFH prior to receiving the first dose of study medication at Treatment Visit T1 per MEDPED (Make Early Diagnosis to Prevent Early Deaths project) criteria by meeting at least one of the following clinical criteria: (a.) Documented diagnosis of HeFH determined by positive genetic testing; or (b.) Documented LDL-C or TC meeting one or more of the following criteria: (i.) LDL-C >200 mg/dL (5.2 mmol/L) or total cholesterol (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
  • Current treatment with approved stable LMTs, 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 patients taking a statin as per co-administration instructions defined in the statin label) Patient 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 patient’s self-reported history of LMT. A patient’s optimal dose of statin is defined as meeting one of the following criteria: a. the highest approved dose of statin prescribed for the age of the patient based on regional practice or local guidelines; or b. less than the highest approved dose of statin, including no statin, prescribed for the age of the patient based on regional practice or local guidelines (including no statin) if: i. the patient 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 patient 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. Patient/parent and investigator attestation to the patient’s unwillingness to attempt and/or physician advice to not attempt additional statin therapy will be recorded.
  • The patient must have a fasting LDL-C level ≥130 mg/dL (3.4 mmol/L) while on sstable LMT as defined in inclusion criteria 5

You likely can't join if

  • The patient has a diagnosis of HoFH or compound HeFH;
  • The patient had an active malignancy, including those requiring surgery, chemotherapy, and/or radiation, in the past 5 years. Nonmetastatic basal or squamous cell carcinoma of the skin and cervical carcinoma in situ are allowed
  • The patient has an unexplained (ie, not associated with recent trauma or physically strenuous activity) serum creatine kinase (CK) value >3 × ULN at any time before randomization. Patients with an explained elevation in serum CK must have single repeat serum CK value ≤3 × ULN before enrollment
  • The patient has a history of drug or alcohol abuse within the last 2 years or is unwilling to refrain from alcohol consumption for the duration of the study, or uses any illicit drugs, or has a history of amphetamine or derivatives abuse or cocaine abuse. Patients who are using amphetamine derivatives prescribed by and who are under the care of a health care practitioner can be enrolled after evaluation by the Investigator
  • The patient has donated blood, undergone multiple blood draws in a clinical study, experienced major trauma, received a blood transfusion, or undergone surgery, with or without blood loss, within 30 days before enrollment
  • The patient has used any experimental or investigational drugs within 30 days before screening and throughout the trial
See the full eligibility criteria
Who can join
  • The patient's parent(s)/guardian(s) must be willing to provide written informed consent and the patient must provide informed assent before any study-specific procedures are performed
  • The patient must be aged 6-17 years old and willing to swallow tablets
  • The patient must weigh at least 16 kg
  • The patient must have a diagnosis of HeFH prior to receiving the first dose of study medication at Treatment Visit T1 per MEDPED (Make Early Diagnosis to Prevent Early Deaths project) criteria by meeting at least one of the following clinical criteria: (a.) Documented diagnosis of HeFH determined by positive genetic testing; or (b.) Documented LDL-C or TC meeting one or more of the following criteria: (i.) LDL-C >200 mg/dL (5.2 mmol/L) or total cholesterol (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
  • Current treatment with approved stable LMTs, 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 patients taking a statin as per co-administration instructions defined in the statin label) Patient 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 patient’s self-reported history of LMT. A patient’s optimal dose of statin is defined as meeting one of the following criteria: a. the highest approved dose of statin prescribed for the age of the patient based on regional practice or local guidelines; or b. less than the highest approved dose of statin, including no statin, prescribed for the age of the patient based on regional practice or local guidelines (including no statin) if: i. the patient 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 patient 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. Patient/parent and investigator attestation to the patient’s unwillingness to attempt and/or physician advice to not attempt additional statin therapy will be recorded.
  • The patient must have a fasting LDL-C level ≥130 mg/dL (3.4 mmol/L) while on sstable LMT as defined in inclusion criteria 5
  • The patient may be male or female. Females must not be pregnant (or planning to become pregnant within 30 days after the last dose of investigational medicinal product [IMP]) breastfeeding and must be sexually inactive or willing to use 1 acceptable method of birth control. The minimal requirement for use of acceptable contraception is from the time the informed consent form (ICF) is signed, during the study period, and for at least 30 days after the last dose of IMP. Acceptable methods of birth control include: a. placement of an intrauterine device (IUD) with or without hormones, b. established use of oral, implanted, topical, or injectable, or hormonal method of contraception associated with inhibition of ovulation, or c. barrier methods, including condom or occlusive cap with spermicidal foam or spermicidal jelly, There are no protocol-specific birth control requirements for males who have partners that can become pregnant.
What rules you out
  • The patient has a diagnosis of HoFH or compound HeFH;
  • The patient had an active malignancy, including those requiring surgery, chemotherapy, and/or radiation, in the past 5 years. Nonmetastatic basal or squamous cell carcinoma of the skin and cervical carcinoma in situ are allowed
  • The patient has an unexplained (ie, not associated with recent trauma or physically strenuous activity) serum creatine kinase (CK) value >3 × ULN at any time before randomization. Patients with an explained elevation in serum CK must have single repeat serum CK value ≤3 × ULN before enrollment
  • The patient has a history of drug or alcohol abuse within the last 2 years or is unwilling to refrain from alcohol consumption for the duration of the study, or uses any illicit drugs, or has a history of amphetamine or derivatives abuse or cocaine abuse. Patients who are using amphetamine derivatives prescribed by and who are under the care of a health care practitioner can be enrolled after evaluation by the Investigator
  • The patient has donated blood, undergone multiple blood draws in a clinical study, experienced major trauma, received a blood transfusion, or undergone surgery, with or without blood loss, within 30 days before enrollment
  • The patient has used any experimental or investigational drugs within 30 days before screening and throughout the trial
  • The patient has previously participated in a clinical study of bempedoic acid
  • The patient is taking any of the following medications or therapies, except as indicated below: a. Mipomersen or lomitapide (current or within 6 months of Screening). b.PCKS9 inhibitors including evolocumab or alirocumab (current or within 3 months of Screening). c. Lipid apheresis (current or within 8 weeks of Screening or intends to have lipidapheresis treatments throughout the trial). d. Systemic corticosteroids (current or within 4 weeks prior to enrollment; topical and inhaled corticosteroids are allowed). e. Red yeast rice extract (also known as monascus purpureus extract or Cholestin) containing products (current or within 4 weeks of Screening); f. Lipid altering nutritional supplements including berberine, psyllium (Metamucil®),green tea extract, sitostanol (found in oral nutritional supplements and some margarines, such as Benecol), beta-sitosterol(found in oral nutritional supplements and some margarines, such as Promise Activ), pantothine and policosanol (current or within 4 weeks of Screening); g. Bile acid sequestrants, fibrates, omega 3 fatty acids, or niacin, unless the dose has been stable for ≥6 weeks and will remain stable throughout the trial. h. Simvastatin >20 mg or pravastatin >40 mg (current or within 4 weeks of Screening).
  • The patient has a history or evidence of any other clinically significant condition, or planned or expected procedure that in the opinion of the Investigator, may compromise the patient's safety or ability to complete the study
  • The patient has a situational (ie, geographical) finding that, in the Investigator's opinion, may compromise the patient's safety or ability to complete the study
  • The patient is an employee or contractor of the facility that is conducting the study or is a family member of the Investigator, subInvestigator, or any Sponsor personnel
  • The patient has a fasting triglyceride (TG) level ≥400 mg/dL (4.5 mmol/L)
  • The patient 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)
  • The patient has liver disease or dysfunction, including: a. positive serology for hepatitis B surface antigen (HBsAg) and/or hepatitis C virus antibodies (HCV-AB), or b. serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value ≥2 × ULN and/or serum total bilirubin (TB) value ≥2 × ULN
  • The patient has renal dysfunction or glomerulonephritis, including an estimated glomerular filtration rate (eGFR) <75 mL/min/1.73 m2 (as determined by the central laboratory using the Revised ["Bedside"] Schwartz formula)
  • The patient has Stage 2 hypertension (based on gender, age and height; see Appendix 4)
  • The patient has a gastrointestinal condition that may affect drug absorption
  • The patient has a history of hematologic or coagulation disorders, anemia, or a hemoglobin (Hgb) level <11.5 g/dL
  • The patient has type 1 or type 2 diabetes, or newly diagnosed impaired glucose tolerance (within 3 months of Screening)

The study team makes the final eligibility decision.

Where it's taking place

  • Canada
  • United States

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Canada; United States. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.