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    Summary
    EudraCT Number:2011-002472-16
    Sponsor's Protocol Code Number:CCD09
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-08-22
    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
    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 ConcernedGermany - BfArM
    A.2EudraCT number2011-002472-16
    A.3Full title of the trial
    Open, prospective, diagnostic, multicentre study in healthy subjects, patients with urea cycle disorders (UCD), and carriers of UCD mutations, to evaluate in vivo ureagenesis measured after a single application of Sodium [1,2-13C]-Acetate
    A.4.1Sponsor's protocol code numberCCD09
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCytonet GmbH & Co KG
    B.1.3.4CountryGermany
    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 organisationCytonet GmbH & Co KG
    B.5.2Functional name of contact pointClinical Study Manager
    B.5.3 Address:
    B.5.3.1Street AddressAlbert-Ludwig-Grimm-Str. 20
    B.5.3.2Town/ cityWeinheim
    B.5.3.3Post code69469
    B.5.3.4CountryGermany
    B.5.4Telephone number06201 / 25 98-130
    B.5.5Fax number06201 / 25 98-28
    B.5.6E-mailursula.friedrichs@cytonet.de
    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 nameSodium [1,2-13C]-Acetate
    D.3.2Product code Sodium [1,2-13C]-Acetate
    D.3.4Pharmaceutical form Powder for oral solution
    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 INNSodium (1,2-13C) acetate
    D.3.9.1CAS number 56374-56-2
    D.3.9.2Current sponsor codeCCD09
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    1. Healthy subjects
    2. Symptomatic UCD patients with genetically confirmed
    CPSD, OTCD, ASSD, or ASLD
    3. Asymptomatic carriers of UCD mutations (e.g. parents)
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level LLT
    E.1.2Classification code 10013373
    E.1.2Term Disorders of urea cycle metabolism
    E.1.2System Organ Class 10027433 - Metabolism and nutrition 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 investigate the performance of the urea cycle in healthy subjects,
    patients with urea cycle disorders (UCD), and carriers of UCD mutations
    by use of the 13C-ureagenesis assay.
    • To compare the performance of the urea cycle with respect to the
    genotype and to the clinical phenotype.
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All study groups:
    • Written informed consent given by subjects or his/her parents/legal guardians who are able to understand and follow instructions related to the study

    Group 1:
    • Age: 18 - 65 years
    • Healthy subjects
    • No clinical or laboratory parameter outside normal ranges at
    screening and judged as clinically relevant by the investigator

    Group 2:
    Age: 0 - 65 years
    • Symptomatic subjects with genetically confirmed
    • Carbamylphosphate synthetase I Deficiency [CPSD]
    • Ornithine Transcarbamylase Deficiency [OTCD]
    • Argininosuccinate Synthetase Deficiency [Citrullinaemia type I]
    • Argininosuccinate Lyase Deficiency [ASLD]
    • at least 1 metabolic decompensation with clinical signs ofhyperammonemia in medical history or genetically confirmed and
    prospectively treated siblings of symptomatic patients, even without clinical symptoms
    • Confirmed diagnosis and medical history available (in particular
    number and severity of metabolic crises)

    Group 3:
    • Age: 0 - 65 years
    • Asymptomatic carriers of mutations for
    • Carbamylphosphate synthetase I Deficiency [CPSD]
    • Ornithine Transcarbamylase Deficiency [OTCD]
    • Argininosuccinate Synthetase Deficiency [Citrullinaemia type 1]
    • Argininosuccinate Lyase Deficiency [ASLD]
    • no dietary protein restriction, no intake of ammonia scavenging drugs, no known metabolic decompensation with clinical signs of
    hyperammonemia
    E.4Principal exclusion criteria
    • Acute illness, including vomiting, fever or other sign of infection
    • Participation in other invasive clinical trials within 30 days prior to
    inclusion
    • Liver or renal disease
    • Acute seizures
    • Coma
    • Bleeding disorder
    • Blood ammonia > 100 μmol/l for patients with a urea cycle disorder and blood ammonia > normal for healthy probands and asymptomatic carriers
    • Metabolic acidosis
    • Pregnancy or lactation
    • Body weight < 8 kg
    E.5 End points
    E.5.1Primary end point(s)
    Formation of 13C-urea in plasma
    E.5.2Secondary end point(s)
    Diagnostic variables (to be assessed retrospectively in UCD subjects)
    • Laboratory parameters: all ammonia and glutamine levels in plasma (in history)
    • History of protein intake (if applicable)
    • Nutritional status (in history)
    • DNA mutation (at study entry or after evaluation for patients that do not provide such data already)
    • Number, duration and severity of metabolic crises (e.g. number of hospitalizations due to crises, maximal ammonia concentration and duration of ammonia elevation, coma yes/no) (in history)
    • Use of ammonia scavenging drugs (in history)
    • Intake of arginine, citrulline (apart from ASSD) and amino acid
    mixture (in history)
    • Neurological status (in history)

    Safety and metabolic variables (to be assessed in all subjects):
    • Vital signs (blood pressure, heart rate, temperature and respiratory rate at enrollment and after completion)
    • Complete blood count without differential (immediately after
    enrollment)
    • Adverse events
    • Ammonia, Amino acids (immediately after enrollment, 0 min), Urea in serum (only 0 min)
    • CRP (immediately after enrollment)
    • Venous lactate and blood gases: pH, pCO2, pO2, bicarbonate
    (immediately after enrollment)
    • Blood glucose (immediately after enrollment and hourly during the 13C assay)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety No
    E.6.5Efficacy No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Evaluation of diagnostic method
    E.7.2Therapeutic exploratory (Phase II) No
    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 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 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months3
    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) 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 Yes
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Could be possible in handicapped UCD patients and children
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children 0-18 years and potentially handicapped
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    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)
    Medical care after the discharge from the study will be provided by the
    patient's general practitioner (Group 2) only. Group 1 and 3 not applicable.
    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 Decision2011-12-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-12-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-07-06
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