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    Summary
    EudraCT Number:2014-003249-82
    Sponsor's Protocol Code Number:HPN-100-012SE
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-02-10
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2014-003249-82
    A.3Full title of the trial
    A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment with HPN-100, in Pediatric Subjects under 6 Years of Age with Urea Cycle Disorders (UCDs)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment with HPN-100, in Pediatric Subjects under 6 Years of Age with Urea Cycle Disorders (UCDs)
    A.4.1Sponsor's protocol code numberHPN-100-012SE
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01347073
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/068/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorHyperion Therapeutics, Inc.
    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 supportHyperion Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHyperion Therapeutics, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address2000 Sierra Point Parkway, Suite 400
    B.5.3.2Town/ cityBrisbane, California
    B.5.3.3Post code94005
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1650745 7851
    B.5.6E-mailbruce.scharschmidt@hyperiontx.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 numberEU/7/10/733
    D.3 Description of the IMP
    D.3.1Product nameGlycerol phenylbutyrate
    D.3.2Product code HPN-100
    D.3.4Pharmaceutical form Oral liquid
    D.3.4.1Specific paediatric formulation Yes
    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 INNGlycerol phenylbutyrate
    D.3.9.1CAS number 611168-24-2
    D.3.9.3Other descriptive nameGLYCEROL PHENYLBUTYRATE
    D.3.9.4EV Substance CodeSUB96043
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.1
    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) 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
    Urea Cycle Disorders (UCDs)
    E.1.1.1Medical condition in easily understood language
    Urea Cycle Disorders (UCDs)
    E.1.1.2Therapeutic area Body processes [G] - Physiological processes [G07]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess safety, pharmacokinetics, and ammonia control in pediatric patients with urea cycle disorders (UCDs) 29 days to < 6 years of age during treatment with HPN 100
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patient aged 29 days to < 6 years old; calculation of the lower age limit began at the corrected gestational age of 40 weeks for patients born prematurely.
    2. Signed informed consent by the patient’s legally acceptable representative
    3. Suspected or confirmed UCD diagnosis of any subtype, except NAGS deficiency
    4. On stable dose of NaPBA powder for at least 5 days before Day 1
    5. Not receiving NaBz for at least 5 days before Day 1
    6. No concomitant illness that would preclude safe participation as judged by the investigator
    7. Able to receive medication orally
    8. Had not undergone liver transplantation, including hepatocellular transplantation
    9. Judged sufficiently stable and compliant with diet and treatment to be suitable for enrollment
    E.4Principal exclusion criteria
    1. Screening ammonia level > 100 μmol/L and signs and symptoms indicative of hyperammonemia; patients may have been rescreened after their ammonia was controlled
    and they were clinically stable, at the discretion of the investigator.
    2. Use of any investigational drug within 30 days of Day 1
    3. Active infection (viral or bacterial) or any other condition that could increase ammonia levels
    4. Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.03, except Grade 3 elevations in ammonia and liver enzymes, defined as levels 5–20 times ULN in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable patient
    5. Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, could have put the patient at increased risk by participating in this study
    6. Known hypersensitivity to PAA or PBA
    7. Liver transplant, including hepatocellular transplant
    8. Currently treated with Carbaglu® (carglumic acid)
    E.5 End points
    E.5.1Primary end point(s)
    Rate of adverse events
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    E.5.2Secondary end point(s)
    • Mean and maximum blood ammonia levels on HPN-100 compared with NaPBA (data collected pre-enrollment and on Day 1)
    • Frequency of hyperammonemic crises on HPN-100 compared with the pre-enrollment period
    • Frequency of ammonia levels > 2 times the upper limit of normal (ULN) on HPN-100 compared with NaPBA (pre-enrollment and on Day 1)
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    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
    Canada
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 23
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 7
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 16
    F.1.1.6Adolescents (12-17 years) No
    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 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 No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    A sample research subject information and consent form was provided to the parents of the child, explaining full details of the proposed trial.
    Parents signed the form if in agreement for their child to participate 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 23
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Long term follow up for a duration of up to 20 months.
    G. Investigator Networks to be involved in the Trial
    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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