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    Summary
    EudraCT Number:2021-000824-36
    Sponsor's Protocol Code Number:HLSC-UCD-01
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-10-01
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-000824-36
    A.3Full title of the trial
    An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in pediatric subjects up to the age of 10 years with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to pediatric subjects without UCD.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial assessing urea formation capacity in children up to 10 years of age using 15N ammonium chloride tracer
    A.3.2Name or abbreviated title of the trial where available
    15N ammonium chloride ureagenesis validation clinical trial
    A.4.1Sponsor's protocol code numberHLSC-UCD-01
    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 SponsorUnicyte AG
    B.1.3.4CountrySwitzerland
    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 supportUnicyte AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUnicyte AG
    B.5.2Functional name of contact pointChief Operations Officer
    B.5.3 Address:
    B.5.3.1Street AddressAawasserstrasse 2
    B.5.3.2Town/ cityOberdorf NW
    B.5.3.3Post code6370
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+49 (173) 366 9051
    B.5.6E-mailc.buck@unicyte.ch
    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 name15N ammonium chloride (15NH4Cl)
    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 INNAmmonium (15N) chloride
    D.3.9.1CAS number 39466-62-1
    D.3.9.2Current sponsor codeAmmonium (15N) chloride
    D.3.9.3Other descriptive nameAmmonium (15N) chloride
    D.3.9.4EV Substance CodeSUB222811
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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 Yes
    D.3.11.13.1Other medicinal product typeStable isotope diagnostic tracer 15N ammonium chloride (15NH4Cl)
    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
    Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS1, ASL, OTC
    Subjects without UCD can have other stable illness that not interfere with the clinical trial according to the investigator judgement
    E.1.1.1Medical condition in easily understood language
    Urea formation disorders
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10080020
    E.1.2Term Urea cycle disorder
    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 assess the intra-subject inter-occasion variability of ureagenesis in pediatric subjects with a confirmed diagnosis of neonatal-onset UCD over a period of up to 36 weeks using a 15NH4Cl diagnostic tracer.
    E.2.2Secondary objectives of the trial
    Key Sec Objs: • To assess the intra-subject inter-occasion variability of ureagenesis in pediatric subjects with a confirmed diagnosis of infantile-onset UCD over a period of up to 36 weeks using a 15NH4Cl diagnostic tracer. •To assess disease stability in pediatric subjects with a confirmed diagnosis of UCD over a period of up to 36 weeks.
    Secondary: •To assess the ureagenesis and its variability in pediatric subjects without UCD over a period of 12 weeks using a 15NH4Cl diagnostic tracer. •To compare the inter-subject variability and extent of impairment of ureagenesis in subjects with UCD with the ureagenesis capacity in pediatric subjects without UCD using a 15NH4Cl diagnostic tracer. •To correlate the ureagenesis, in pediatric subjects with and without UCD, as determined by the 15NH4Cl tracer assay, with blood levels of ammonia, citrulline and glutamine
    Safety Objs: To assess the safety and tolerability of 15NH4Cl diagnostic tracer in pediatric subjects with and without UCD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A subject must meet ALL of the following criteria at Screening to be eligible for this trial, with the exception of criteria 1 and 2 which do not apply to subjects without UCD and criterium 3 that only applies for subjects without UCD:
    Only for subjects with UCD
    1. Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS1, ASL, OTC. Note: All subjects should have genotyping information available, however if an exact genetic diagnosis is not available, diagnosis of the UCD sub-type may be confirmed by well accepted biochemical parameters
    2. Subject has neonatal or infantile onset of UCD signs and symptoms within the first 12 months of life; or subjects who have a family history of UCD and are asymptomatic after birth due to a therapeutic regimen started directly after birth;
    Only for subjects without UCD
    3. Subjects without UCD can have other stable illness that does not interfere with the clinical trial according to the investigator judgement;
    For all subjects (with and without UCD)
    4. Male and female subjects aged up to 10 years, inclusive;
    5. Subject has a body weight within the 5-95 percentile of the corresponding age according to the CDC Growth Charts;
    6. Subject has stable clinical conditions (any acute condition needs to be stabilised/treated before inclusion);
    7. The parent(s) / legal representative(s) agrees that the subject will not participate in any interventional clinical trial with an investigational drug suspected of having an interaction with the urea cycle or 15NH4Cl diagnostic tracer for the duration of the trial until the final follow-up telephone call;
    8. Ability and willingness of the parent(s) / legal representative(s) to comply with the protocol requirements, including ability to bring the subject to the scheduled trial visits;
    9. Written informed consent by the parent(s) / legal representative(s) of the subject and assent from the pediatric subject when required by local regulations, the IRB/IEC or trial site.

    E.4Principal exclusion criteria
    A subject must meet NONE of the following criteria at Screening to be eligible for this trial, with the exception of criterion 1 which does not apply to subjects with UCD:
    Only for subjects with UCD
    1. Subject has any suspected UCD of any sub-type. Note: subjects suspected of having a UCD of any sub-type, but without either confirmatory genotyping information or a typical biochemical diagnostic pattern for any UCD gene defect, will not be enrolled in this trial.
    For all subjects (with and without UCD)
    2. Subject is a premature neonate (up to 37 gestation weeks not completed);
    3. Subject is in a period of significant post-natal weight drop based on the judgement of the investigator ;
    4. Subject has received any investigational compound within 30 days (or 5 half-lives, whichever is longer) prior to first dose of diagnostic tracer and according to the investigator judgement could interfere with the clinical trial;
    5. Subject has any other acute severe / other genetic / life limiting disorder that would interfere with ethical and/or medical standards in the conduct or follow up of the trial.
    6. Subject has acute liver failure, clinical or radiological evidence of liver fibrosis or cirrhosis, or presents a hepatic or extrahepatic malignancy.
    E.5 End points
    E.5.1Primary end point(s)
    •The intra-subject inter-occasion variability of the [15N] urea enrichment area under the plasma concentration-time curve over 2 hours (AUC0-2) following up to 4 15NH4Cl diagnostic tracer administrations over a 36-week period to pediatric subjects with neonatal-onset UCD.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Over a 36-week period
    E.5.2Secondary end point(s)
    Key Secondary Endpoints:
    •The intra-subject inter-occasion variability of the [15N] urea enrichment area under the plasma concentration-time curve over 2 hours (AUC0-2) following up to 4 15NH4Cl diagnostic tracer administrations over a 36-week period to pediatric subjects with infantile-onset UCD.
    •The number of pediatric subjects with UCD demonstrating stable disease at Weeks 0, 12, 24 and 36.
    •The blood concentrations of ammonia, glutamine and citrulline up to 2 hours following
    o up to 4 15NH4Cl diagnostic tracer administrations to pediatric subjects with UCD (Weeks 0, 12, 24 and 36).
    o up to 2 15NH4Cl diagnostic tracer administrations to pediatric subjects without UCD (Weeks 0 and 12).
    •The number of hyperammonaemia events and crises experienced since the previous trial visit at Weeks 12, 24 and 36 (subjects with UCD only).
    •The number of pediatric subjects following a stable protein diet, with no unplanned adjustments since the previous trial visit, at Weeks 12, 24 and 36 (subjects with UCD only).
    Secondary Endpoints:
    •The intra-subject inter-occasion variability of the [15N] urea enrichment area under the plasma concentration-time curve over 2 hours (AUC0-2) following
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week period to pediatric subjects with infantile-onset UCD.
    o up to 2 15NH4Cl diagnostic tracer administrations over a 12-week period to pediatric subjects without UCD.
    •The inter-subject variance of the [15N] urea enrichment area under the plasma concentration-time curve over 2 hours (AUC0-2) following each 15NH4Cl diagnostic tracer administration
    o to pediatric subjects with neonatal-onset UCD (Weeks 0, 12, 24 and 36).
    o to pediatric subjects with infantile-onset UCD (Weeks 0, 12, 24 and 36).
    o to pediatric subjects without UCD (Weeks 0 and 12).
    •The inter-subject inter-occasion variability of the [15N] urea enrichment area under the plasma concentration-time curve over 2 hours (AUC0-2) following
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week period to pediatric subjects with neonatal-onset UCD.
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week period to pediatric subjects with infantile-onset UCD.
    o up to 2 15NH4Cl diagnostic tracer administrations over a 12-week period to pediatric subjects without UCD.
    •The inter-subject variance of the area under the blood concentration-time curve over 2 hours (AUC0-2) for ammonia, citrulline and glutamine following
    o up to 4 15NH4Cl diagnostic tracer administrations over a 36-week period to pediatric subjects with UCD.
    o up to 2 15NH4Cl diagnostic tracer administrations over a 12-week period to pediatric subjects without UCD.
    Safety Endpoints:
    •Adverse events (AEs); physical, neurological and child development examination findings; vital signs; clinical laboratory tests; biochemical parameters; plasma amino acid levels; frequency and severity of hyperammonaemia events and crises; and changes in protein intake and concomitant medication.
    E.5.2.1Timepoint(s) of evaluation of this end point
    According to the protocol
    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 Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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
    Ureagenesis assessment using 15N labelled ammonium chloride (15NH4Cl)
    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 Yes
    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 Yes
    E.8.2.3.1Comparator description
    Subjects without a diagnosis or suspicion of UCD will be enrolled in this trial as control subjects
    E.8.2.4Number of treatment arms in the trial1
    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 EEA7
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    Saudi Arabia
    Austria
    France
    Spain
    Switzerland
    Germany
    Italy
    Belgium
    Turkey
    United Kingdom
    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.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months10
    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 months11
    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.1.1Number of subjects for this age range: 30
    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.1.1.3.1Number of subjects for this age range: 10
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    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 Yes
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 30
    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)
    No diagnostic tracer will be provided after the end of the trial.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-10-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-02-13
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