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    Summary
    EudraCT Number:2005-003511-75
    Sponsor's Protocol Code Number:WEL-410
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2006-07-27
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2005-003511-75
    A.3Full title of the trial
    Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Colesevelam HCl Administered to Pediatric Patients with Heterozygous Familial Hypercholesterolemia on a Stable Dose of Statins or Treatment Naïve to Lipid-Lowering Therapy
    A.4.1Sponsor's protocol code numberWEL-410
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorDaiichi Sankyo Pharma Development
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameColesevelam HCl; WelChol
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNcolesevelam HCl
    D.3.9.3Other descriptive nameCholestagel
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number625
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pediatric Heterozygous Familial Hypercholesterolemia (heFH)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 8.1
    E.1.2Level LLT
    E.1.2Classification code 10057079
    E.1.2Term Heterozygous familial hypercholesterolemia
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this trial is to evaluate the lipid lowering efficacy and safety of colesevelam HCl therapy administered to heFH pediatric patients 10 through 17 years of age who are on a stable dose of a pediatric-approved statin monotherapy (atorvastatin, lovastatin, simvastatin, or pravastatin), or who are treatment naïve to lipid-lowering therapy. For Period II (Day 1 to Week 8): To evaluate the low density lipoprotein cholesterol (LDL-C) lowering efficacy of colesevelam HCl administered to heFH patients on a stable dose of statins or who are treatment naïve to lipid lowering therapy.
    E.2.2Secondary objectives of the trial
    For Period III (Week 8 to Week 26): To evaluate the long-term safety and efficacy of high dose colesevelam HCl administered to heFH patients while optimizing statin therapy for treatment to goal.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent from the patient’s parent or guardian and assent from the patient. (Consent form is approved by the institutional review board (IRB) or other committee).

    2. Male or female patient is 10 through 17 years of age.

    3. Patient has a diagnosis of heFH defined as a known mutation in a copy of the patient’s LDL-receptor or apolipoprotein B (Apo B) gene. In the absence of a genetic diagnosis the patient must have the following LDL-C criteria to be classified as a heFH patient:
    • A documented history of untreated LDL-C >160 mg/dL (4.14 mmol/L) In combination with at least one of the following:
    • The history or presence, in the patient or a first degree relative, of a documented tendinous or cutaneous xanthoma or premature (before age 45 years) corneal arcus; or
    • The history or presence, in an adult first-degree relative or biological offspring of a documented mutation in a copy of the individual’s (relative or offspring) LDL-receptor or apolipoprotein B (Apo B) gene; or
    • The presence, in an adult first-degree relative, of documented untreated LDL-C >190 mg/dL (4.9 mmol/L), or sibling <18 years old with LDL-C >160 mg/dL (4.14 mmol/L); or
    • A documented history, in a first-degree relative, of premature coronary artery disease or sudden death from natural causes prior to age 55 years if male, or prior to age 60 years of age if female.

    4. Patient has LDL-C >130 mg/dL (3.37 mmol/L) while on statin therapy, or LDL-C >160 mg/dL (4.14 mmol/L) naïve to hypolipidemic therapy and diet stabilized.

    5. Patient must be stable on a statin plus diet for 6-weeks; or for lipid-lowering treatment naïve patient, stable on a NCEP Step 1 diet or equivalent for 4 weeks prior to Visit 1.

    6. Triglycerides (TG) at Visit 1 <250 mg/dL (2.83 mmol/L).

    7. Patients should be of at least Tanner Stage 2.

    8. Female patients may enroll if all 5 of the following criteria are met:
    • At least 1 year post-menarche
    • Negative urine pregnancy test at screening
    • Not lactating
    • Do not plan to become pregnant during the study
    • Use appropriate contraceptive methods if sexually active. If the patient is on oral contraceptives, it needs to be taken at least 3 months prior to randomization.

    9. Mean treatment compliance during Period I (Visit 2 and Visit 3) must be ≥75%.
    E.4Principal exclusion criteria
    1. Patients unable or unwilling to take the study drug.

    2. Secondary hyperlipidemia (e.g., due to biliary cirrhosis, dietary abnormalities, nephrotic syndrome, hypothyroidism, etc.).

    3. Fasting TG >250 mg/dL (2.83 mmol/L).

    4. Homozygous familial hypercholesterolemia.

    5. Patients with a history of dysphagia, swallowing disorders, intestinal motility disorders.

    6. Hypertension due to renal disease.

    7. Untreated thyroid disease.

    8. Clinically important liver or renal disorder, or vasculitis. Patients with mild to moderate liver steatosis, per investigator assessment, may be included.

    9. Poorly controlled Type 1 or Type 2 diabetes mellitus defined as HbA1c >9.0%. Diabetics must have documentation of HbA1c within 3 months of the screening visit. Undocumented patients must have their HbA1c assessed at Visit 1. Note: Patients with Type 1/Type 2 diabetes controlled with insulin and/or oral hypoglycemic agents on a stable dose for last 30 days prior to Visit 1 may be included.

    10. Patients who require or are taking any concomitant medication, which may interfere with the objectives of the study. Use of oral anticoagulants or digoxin, unless the dose has been stable for 4 weeks prior to Visit 1 and regular clinical laboratory monitoring is performed.

    11. Screening laboratory values within the following age/gender appropriate reference ranges as assessed by the central laboratory:
    • Hemoglobin
    Male <10 g/dL
    Female <9 g/dL
    • Alanine Transaminase (ALT) (SGPT) >2 x Upper Limit Normal(ULN) for age
    • Aspartate Transaminase (AST) (SGOT) >2 x ULN for age
    • Alkaline Phosphatase >2 x ULN for age
    • Total bilirubin >1 x ULN (unless elevations due to congenital bilirubin metabolism defect, i.e. Gilbert Syndrome)
    • Creatine Phosphokinase (CPK) >5 x ULN for gender unless explained by exercise
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint for this trial in pediatric patients with heFH is that after 8 weeks of treatment (Period II), all patients receiving low dose (1875 mg) and high dose (3750 mg) colesevelam HCl will demonstrate superior lipid-lowering efficacy compared with patients treated with placebo as assessed by percent change from baseline in LDL-C reduction.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Information not present in EudraCT
    E.6.2Prophylaxis Information not present in EudraCT
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Information not present in EudraCT
    E.6.7Pharmacodynamic Information not present in EudraCT
    E.6.8Bioequivalence Information not present in EudraCT
    E.6.9Dose response Information not present in EudraCT
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2006-07-27. Yes
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 200
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2006-11-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2006-10-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2007-12-18
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