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    Summary
    EudraCT Number:2012-005340-24
    Sponsor's Protocol Code Number:SONIA1
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-01-09
    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
    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 ConcernedUK - MHRA
    A.2EudraCT number2012-005340-24
    A.3Full title of the trial
    An international, multicentre, randomised, open-label, no-treatment controlled, parallel group, dose-response study to investigate the effect of once daily nitisinone on 24-hour urinary homogentisic acid excretion in patients with alkaptonuria after 4-weeks treatment.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Suitability of nitisinone in alkaptonuria (AKU).
    A.3.2Name or abbreviated title of the trial where available
    SONIA 1
    A.4.1Sponsor's protocol code numberSONIA1
    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 SponsorUniversity of Liverpool (UniLiv)
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportUniversity of Liverpool
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRoyal Liverpool Unversity Hospital (RLUH)
    B.5.2Functional name of contact pointPrincipal Investigator
    B.5.3 Address:
    B.5.3.1Street AddressPrescot Street
    B.5.3.2Town/ cityLiverpool, Merseyside
    B.5.3.3Post codeL7 8XP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number00441517062000
    B.5.6E-maillrang@liverpool.ac.uk
    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/3/02/096
    D.3 Description of the IMP
    D.3.1Product nameOrfadin 4mg/ml
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    Alkaptonuria (AKU) - a serious, autosomal recessive, multisystem disorder
    E.1.1.1Medical condition in easily understood language
    Alkaptonuria (AKU) - a serious, autosomal recessive, multisystem disorder. Also, known as 'black bone' disease.
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    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 investigate the effect of different doses of once daily nitisinone on 24-hour urinary homogentisic acid excretion (u-HGA24) in patients with alkaptonuria after 4-weeks treatment.
    E.2.2Secondary objectives of the trial
    • To investigate the effect of different doses of once daily nitisinone on plasma homogentisic acid concentration (p-HGA)
    and plasma tyrosine concentration (p-Tyr) in patients with alkaptonuria.
    • To determine the pharmacokinetics (PK) of nitisinone at steady state and to test for PK dose-proportionality.
    • To describe the relationship between PK variables of nitisinone, u-HGA24, p-HGA and p-Tyr.
    • To assess the safety of nitisinone at doses relevant for the treatment of alkaptonuria.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of AKU verified by previously documented elevated urinary homogentisic acid excretion.
    2. Age ≥18 years.
    3. Willing and able to visit the investigational site for study visits.
    4. Signed written informed consent given.

    E.4Principal exclusion criteria
    1. Currently pregnant or lactating.
    2. Female patient of child-bearing potential not using a reliable method of contraception.
    3. Known allergy to nitisinone or any of the constituents of the investigational product.
    4. Current keratopathy or uncontrolled glaucoma.
    5. Current malignancy.
    6. Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
    7. Unstable cardiovascular disease.
    8. Serum potassium < 3.0 mmol/L.
    9. eGFR < 60 mL/min.
    10. ALT > 3 x upper limit of normal.
    11. Hemoglobin < 10.0 g/dL.
    12. Platelets < 100 x 109/L.
    13. White blood count < 3.0 x 109/L.
    14. History of alcohol or drug abuse.
    15. Participation in another clinical study within 3 months of randomization.
    16. Treatment with nitisinone within 60 days of randomization.
    17. Psychiatric illness or neurological disease that interferes with compliance or communication with health care personnel.
    18. Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
    19. Foreseeable inability to cooperate with given instructions or study procedures.
    E.5 End points
    E.5.1Primary end point(s)
    u-HGA24 after 4 weeks of treatment with nitisinone.
    E.5.1.1Timepoint(s) of evaluation of this end point
    A follow-up phone call will take place 2 weeks (+/- 3 days) after the Final Visit. The patient will be questioned about concomitant medication and adverse events.
    E.5.2Secondary end point(s)
    • u-HGA24 after 1, 2 and 3 weeks of treatment with nitisinone.
    • Proportion of patients achieving normal levels of u-HGA24 at weeks 1, 2, 3 and 4.
    • p-HGA and p-Tyr at Weeks 1, 2, 3, and 4.
    • 24-hour plasma HGA and plasma tyrosine profiles at baseline and Week 4.
    • Nitisinone steady-state pharmacokinetic variables.
    • Adverse events, clinical chemistry and haematology, vital signs, ECG.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visit 2: The 24-hour urine collection will be done at home. Patients should begin collection in the morning, immediately after taking the daily dose of study medication. Collection ends the following morning at the same time.
    Visit 3: As for Visit 1. The patient will take the last dose of study medication immediately before the 24-hour collection period begins.
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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
    No treatment control group
    E.8.2.4Number of treatment arms in the trial5
    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 EEA2
    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
    LVLS and one follow-up telephone call.
    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 days0
    E.8.9.2In all countries concerned by the trial months3
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 37
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 40
    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)
    Back to usual care
    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 Decision2013-02-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-01-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-12-13
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