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    Summary
    EudraCT Number:2014-003246-26
    Sponsor's Protocol Code Number:HPN-100-005SO
    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 number2014-003246-26
    A.3Full title of the trial
    A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN 100 Compared to Sodium Phenylbutyrate in Children 6–17 Years of Age with Urea Cycle Disorders, with a Long-Term Safety Extension
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN 100 Compared to Sodium Phenylbutyrate in Children 6–17 Years of Age with Urea Cycle Disorders, with a Long-Term Safety Extension
    A.4.1Sponsor's protocol code numberHPN-100-005SO
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00947544
    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 Trials 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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name AMMONAPS®
    D.2.1.1.2Name of the Marketing Authorisation holderSwedish Orphan Biovitrum International AB
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and pharmacokinetic (PK) characteristics of HPN 100 compared with NaPBA in pediatric patients with urea cycle disorders (UCDs)
    E.2.2Secondary objectives of the trial
    To evaluate control of blood ammonia by HPN-100 compared with NaPBA in pediatric patients with UCDs.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female patients 6–17 years old
    2. Signed informed consent by patient’s legally acceptable representative and assent by patient, as applicable
    3. Diagnosis of urea cycle disorder (enzyme or transporter deficiency) confirmed via enzymatic, biochemical, or genetic testing
    4. Patients were on a stable dose of NaPBA for a diagnosis of UCD for at least 1 week prior to the Day 1 visit. Patients who were not on a stable dose of NaPBA at the initial screening visit may have been converted to a stable dose of NaPBA during the screening period and enrolled as long as they were on a stable dose of NaPBA for at least 1 week prior to Day 1.
    5. Able to perform and comply with study activities, including blood draws and urine collections
    6. Negative pregnancy test for all females of childbearing potential
    7. All females of childbearing age and all sexually active males must have agreed to use an acceptable method of contraception throughout the study. Appropriate contraceptive methods included hormonal contraceptives (oral, injected, implanted, or transdermal), tubal ligation, intrauterine device, hysterectomy, vasectomy, or double-barrier methods. Abstinence was an acceptable form of birth control, though appropriate contraception must have been used if the patient became sexually active.
    E.4Principal exclusion criteria
    1. Screening ammonia level of ≥ 100 μmol/L or signs and symptoms indicative of hyperammonemia; patients may have been rescreened after their ammonia was controlled, at the discretion of the investigator
    2. History of 4 or more hyperammonemic events (defined as clinical symptoms associated with ammonia of ≥ 100 μmol/L) in the preceding 12 months
    3. Use of any investigational drug within 30 days of Day 1
    4. Active infection (viral or bacterial) or any other condition that may have increased ammonia levels
    5. Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v3.0, except Grade 3 elevations in 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
    6. Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may have put the patient at increased risk by participating in this study
    7. Use of any medication known to significantly affect renal clearance (e.g., probenecid) or to increase protein catabolism (e.g., corticosteroids), or other medication known to increase ammonia levels (e.g., valproate), within the 24 h prior to Day 1 and throughout the study
    8. History of QTc interval prolongation or QTc interval > 450 msec at screening or baseline
    9. Known hypersensitivity to PAA or PBA
    10. Liver transplant, including hepatocellular transplant
    11. Currently treated with sodium benzoate or Carbaglu® (carglumic acid) − At the discretion of the investigator, patients on sodium benzoate who were otherwise eligible to participate may have been switched to 100% NaPBA during the 30-day screening period as part of the study, and at least 7 days prior to Day 1 (Visit 2).
    12. Breastfeeding or lactating females
    E.5 End points
    E.5.1Primary end point(s)
    Rate of adverse events in each treatment group.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Last day of treatment
    E.5.2Secondary end point(s)
    • Blood ammonia AUC0-24on NaPBA vs. HPN-100 on the last day of treatment with each drug
    • Blood ammonia Cmax on NaPBA vs. HPN-100 on the last day of treatment with each drug
    • Average ammonia values on NaPBA vs. HPN-100 on the last day of treatment with each drug
    • U-PAGN values on NaPBA vs. HPN-100
    • Plasma PBA, PAA, and PAGN values on NaPBA vs. HPN-100
    • Rate (percentage) of ammonia values above ULN on NaPBA vs. HPN-100
    E.5.2.1Timepoint(s) of evaluation of this end point
    Last day of treatment
    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
    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: 11
    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.1.1.5.1Number of subjects for this age range: 7
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    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 11
    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 Safety Extension
    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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