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    Summary
    EudraCT Number:2015-005536-17
    Sponsor's Protocol Code Number:UX007G-CL301
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-05-16
    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 ConcernedFrance - ANSM
    A.2EudraCT number2015-005536-17
    A.3Full title of the trial
    A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
    Étude croisée de phase 3, randomisée, contrôlée par placebo et en double insu, visant à évaluer l’efficacité et la sécurité d’emploi de UX007 dans le traitement des troubles du mouvement liés à un syndrome de déficit en transporteur de glucose de type 1 (syndrome de déficit en Glut1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
    A.4.1Sponsor's protocol code numberUX007G-CL301
    A.5.4Other Identifiers
    Name:EMA/190573Number:Unique Product Identifier (UPI)
    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 SponsorUltragenyx Pharmaceutical 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 supportUltragenyx Pharmaceutical Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmaceutical Inc
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato
    B.5.3.3Post codeCA 94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1415483 8813
    B.5.6E-mailUX007G-CL301@ultragenyx.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/3/15/1495
    D.3 Description of the IMP
    D.3.1Product nameUX007
    D.3.2Product code UX007
    D.3.4Pharmaceutical form 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 INNNot available
    D.3.9.1CAS number 620-67-7
    D.3.9.2Current sponsor codeUX007
    D.3.9.3Other descriptive nameTRIHEPTANOIN
    D.3.9.4EV Substance CodeSUB129584
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.96
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Glucose Transporter Type 1 deficiency syndrome
    Syndrome de déficit en transporteur de glucose de type 1
    E.1.1.1Medical condition in easily understood language
    Glucose transporter type 1 (Glut1) deficiency syndrome is a rare genetic metabolic disorder characterized by deficiency of a protein that is required for glucose to cross the blood-brain barrier
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10061032
    E.1.2Term Carbohydrate transport disorder
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary Objectives:
    •Evaluate the efficacy and safety of UX007 in the treatment of disabling paroxysmal movement disorders associated with Glut1 DS
    L’objectif principal de l’étude est le suivant :
    •Évaluer l’efficacité et la sécurité d’emploi de UX007 dans le traitement des troubles du mouvement paroxystiques handicapants associés au syndrome de déficit en Glut1
    E.2.2Secondary objectives of the trial
    Evaluate the efficacy of UX007 compared to placebo, as measured by:
    •Walking capacity and endurance, as determined by the distance walked in 12 minutes during the 12 Minute Walk Test (12MWT)
    •Health-related quality of life assessing physical function, mobility, upper extremity function, fatigue, pain and social health using a PROMIS®-based questionnaire
    •Patient/caregiver global impression of change in clinical status using the Clinical Global Impression – Improvement (CGI-I)
    •Duration of disabling paroxysmal movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily electronic Glut1 DS symptom diary
    •Cognitive function as measured by the Cambridge Neuropsychological Test Automated Battery (CANTAB) (assessed at select sites)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1)Diagnosis of Glut1 DS confirmed by SLC2A1 mutation
    2)Males and females, aged ≥6 years old at the time of informed consent
    3)At least 8 disabling paroxysmal movement disorder events in the 12 weeks prior to the Screening, by subject or caregiver report
    OR
    At least 6 disabling paroxysmal movement disorder events in any 6 consecutive week period, over the last 12 week period prior to the Screening, by subject or caregiver report

    4)At least 4 disabling paroxysmal movement disorder events in 6 week Run-in Period, reported in the daily electronic Glut1 DS symptom diary
    5)≥80% compliance with daily electronic Glut1 DS symptom diary completion during the Run-in Period
    6)Not on KD, modified KD, or ketosis-inducing modified-fat diet for at least 3 months prior to Screening
    7)Plasma level of beta-hydroxybutyrate (BHB) ≤ 1 mmol/L (non-fasting) at Screening
    8)Provide written or verbal assent (if possible) and written informed consent by the patient (if an adult), or by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
    9)Must, in the opinion of the Investigator, be willing and able to complete key aspects of the study and be likely to complete the 22-week, placebo-controlled, treatment period
    10)Patient (or caregiver) must, in the opinion of the Investigator, be able to comply with accurate completion of the study daily electronic Glut1 DS symptom diary
    11)Females of child-bearing potential must have a negative urine pregnancy test at Screening and Baseline and be willing to have additional pregnancy tests during the study. Females considered not to be of childbearing potential include those who have not experienced menarche, are post-menopausal (defined as having no menses for at least 12 months without an alternative medical cause) or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
    12)Participants of child‐bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly effective method of contraception as determined by the site Investigator from the period following the signing of the informed consent through 30 days after last dose of study drug
    E.4Principal exclusion criteria
    1)Any known hypersensitivity to triheptanoin or safflower oil that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
    2)Prior use of triheptanoin within 30 days prior to Screening
    3)History of, or current suicidal ideation, behavior and/or attempts per C-SSRS at Screening or Baseline
    4)Pregnant and/or breastfeeding an infant at Screening or Baseline
    5)Participants unwilling or unable to discontinue use of a prohibited medication or other substance that may confound study objectives (Section 7.4.6.1 [MCT oil, barbiturates, pancreatic lipase inhibitors, KetoCal or other KD supplements, and/or KD])
    6)Glut1 DS treatment regimen, including AEDs, should be stable for at least 30 days prior to Screening
    7)Use of any investigational product (drug, medical food, or supplement, including medium chain triglyceride [MCT] oil, including coconut oil) within 30 days prior to Screening
    8)Has a concurrent disease or condition, or laboratory abnormality that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduces additional safety concerns
    9)Feeding or nutrition that, in the opinion of the dietitian, potentially affects consistent administration of study drug
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the frequency of disabling paroxysmal movement disorders captured as movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily Glut1 DS symptom diary.
    Critère d’évaluation principal
    Le critère d’évaluation principal est la fréquence des troubles du mouvement paroxystiques handicapants mesurés par les événements de trouble du mouvement observés pendant la période d’entretien du traitement, comme consigné dans le journal quotidien des symptômes du syndrome de déficit en Glut1 par le patient/l’aidant.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Daily Glut1 Symptom Diary review: Weeks 0,2,4,6,10,12,14,16,18,22,26,30,34,46,58,70,82,94,106,118,130,142,154,178
    E.5.2Secondary end point(s)
    •Walking capacity and endurance, as determined by the distance walked in 12 minutes during the 12 Minute Walk Test (12MWT)
    •Health-related quality of life assessing physical function, mobility, upper extremity function, fatigue, pain and social health using a PROMIS®-based questionnaire
    •Patient/caregiver global impression of change in clinical status using the Clinical Global Impression – Improvement (CGI-I)
    •Duration of disabling paroxysmal movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily electronic Glut1 DS symptom diary
    •Cognitive function as measured by the Cambridge Neuropsychological Test Automated Battery (CANTAB) (assessed at select sites)
    •Capacité de marche et endurance, comme déterminé par la distance de marche en 12 minutes pendant le test de marche de 12 minutes (TM12M)
    •Qualité de vie liée à la santé évaluant la fonction physique, la mobilité, la fonction des extrémités supérieures, la fatigue, la douleur et la santé sociale au moyen d’un questionnaire basé sur PROMIS®
    •Impression globale de changement de l’état clinique signalée par le patient/l’aidant au moyen de l’échelle d’impression clinique globale - amélioration (Clinical Global Impression - Improvement, CGI-I)
    •Durée des événements de trouble du mouvement paroxystiques handicapants observés pendant la période d’entretien du traitement, comme consigné dans le journal quotidien électronique des symptômes du syndrome de déficit en Glut1 par le patient/l’aidant
    •Fonction cognitive comme mesuré par le test neuropsychologique de Cambridge à batterie automatique (Cambridge Neuropsychological Test Automated Battery, CANTAB) (évaluée dans certains centres sélectionnés)
    E.5.2.1Timepoint(s) of evaluation of this end point
    12MWT: Weeks 0,10,12,22
    PROMIS: Weeks 0,10,12,22,26,30,34,46,58,70,82,94,106,118,142,154,178
    Patient/Caregiver CGI-I: Weeks 10,12,22
    Duration of disabling paroxysmal movement disorders:Weeks 0,2,4,6,10,12,14,16,18,22,26,30,34,46,58,70,82,94,106,118,130,142,154,178
    CANTAB: Weeks 0,10,12,22
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    open-label Extension Period after the initial 22-week double-blind period
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA11
    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
    Australia
    France
    Germany
    Ireland
    Israel
    Italy
    Netherlands
    Spain
    United Kingdom
    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
    Last patient last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 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.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric subjects
    patients pédiatriques
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    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)
    After the initial 22-week double-blind Treatment Period, the open-label Extension Period will begin, wherein all subjects will be treated with UX007 through Week 178 of the study.
    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 Decision2017-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-10-09
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