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    Summary
    EudraCT Number:2011-004528-36
    Sponsor's Protocol Code Number:CAC-002-01
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2011-10-06
    Trial 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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2011-004528-36
    A.3Full title of the trial
    An open-label, single center, non-randomized, continuation study of cholic acid capsules in subjects with inborn errors of bile acid synthesis.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Continuation of cholic acid treatment for people with bile acid disorders who have previously taken part in clinical trials of cholic acid capsules.
    A.4.1Sponsor's protocol code numberCAC-002-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01438411
    A.5.4Other Identifiers
    Name:CCHMC continuation studyNumber:CAC-002-01
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/206/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAsklepion Pharmaceuticals, LLC
    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 supportAsklepion Pharmaceuticals, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAsklepion Pharmaceuticals, LLC
    B.5.2Functional name of contact pointGary R. Pasternack
    B.5.3 Address:
    B.5.3.1Street Address729 East Pratt Street, Suite 360
    B.5.3.2Town/ cityBaltimore, Maryland
    B.5.3.3Post code21202
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1410545 0494
    B.5.5Fax number+1410545 0584
    B.5.6E-mailgary.pasternack@asklepionpharm.com
    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 Yes
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCholic Acid 50 mg and 250 mg Capsules
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    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 INNCholic Acid
    D.3.9.1CAS number 81-25-4
    D.3.9.3Other descriptive nameCHOLIC ACID
    D.3.9.4EV Substance CodeSUB13348MIG
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    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 No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    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 No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with inborn errors of bile acid synthesis and metabolism
    E.1.1.1Medical condition in easily understood language
    Patients with birth defects inherited from their parents which means that they cannot make normal bile acids.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level PT
    E.1.2Classification code 10070882
    E.1.2Term Inborn error in primary bile acid synthesis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the therapeutic efficacy of commercial cholic acid capsules, hard.
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of cholic acid
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients completing protocol CAC-001-01 or transitioned from protocol 91-10-10 or children with newly diagnosed inborn errors of bile acid synthesis.
    Any new, additional infants, children, and adolescents presenting for evaluation of cholestasis defined as a conjugated bilirubin > 2mg/dl or evaluation chronic liver disease, fat and fat soluble vitamin deficiency, growth failure or other signs or symptoms consistent with intestinal intraluminal bile acid deficiency. Patients will have undergone thorough evaluation to define the etiology of cholestasis using conventional screening studies (urine culture α-1-antitrypsin pheno¬type, endocrine studies, STORCH titers, thyroid function tests, liver ultrasound, liver "function" tests and percutaneous liver biopsy).
    Older patients of any age with chronic liver disease if urine screens indicate that they have inborn errors of bile acid metabolism.
    E.4Principal exclusion criteria
    None
    E.5 End points
    E.5.1Primary end point(s)
    Therapeutic effect of commercial cholic acid capsules on:
    - serum transaminases and
    -suppresson of bile acid synthesis of atypical bile acids, as measured in urine and serum bile acid analysis using mass spectrometry.
    -
    E.5.1.1Timepoint(s) of evaluation of this end point
    Liver biochemistries (serum total/direct bilirubin, ALT, AST, total protein, albumin, alkaline phosphatase, GGT and prothrombin time with INR (optional) as a part of standard of care by the attending physician and serum and urine for bile acid analysis by GC-MS, LC-MS and LSIMS will be performed every one month for the first three months, and then every 3-6 months based upon clinical condition of the patient for the next-12 months after starting therapy.
    Thereafter, adjustments in cholic acid dose will be made based upon urinary FAB analysis and frequency of repeat urine FAB-MS analysis will be performed as needed at least annually.
    Liver biochemistries and urine LSIMS will be performed at least annually as part of standard of care for patients with chronic liver disease.
    E.5.2Secondary end point(s)
    Safety and tolerability of TBM cholic acid capsules, as assessed by vital signs, physical examination findings, clinical laboratory results and the incidence and severity of adverse events, compared with baseline data.
    Assessment of malabsorption: height/weight gain, normalisation of steatorrhoea, measurements of vitamin A,E and D and prothrombin time.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Every one month for the first 3 months and then 3-6 months based on the clinical condition of the patient for the next 12 months after starting therapy. Thereafter perfomed at least annually.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    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
    December 2011
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial months9
    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) Yes
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.5Children (2-11years) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    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 No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Newborns (0-27 days), infants and toddlers (28 days to 23 months) and children (2-11 years) included in the study
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 30
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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