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    Summary
    EudraCT Number:2013-003771-35
    Sponsor's Protocol Code Number:UX007G-CL201
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-12-11
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2013-003771-35
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Adaptive Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome
    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 Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
    A.4.1Sponsor's protocol code numberUX007G-CL201
    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 pointJulia Martinisi
    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+1415827 4123
    B.5.6E-mailjmartinisi@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTriheptanoin
    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 % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    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] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level HLGT
    E.1.2Classification code 10039911
    E.1.2Term Seizures (incl subtypes)
    E.1.2System Organ Class 10029205 - Nervous system disorders
    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 of UX007 compared to placebo, between Weeks 2 and 8 of treatment, as measured by the reduction from baseline in frequency of generalized or partial-onset seizures
    • Evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG)
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    • Seizure response rate, defined as the percentage of subjects with at least 50% reduction from baseline in generalized or partial-onset seizures
    • Change from baseline in frequency of seizure activity as measured by electroencephalography (EEG) abnormalities
    • Change from baseline in cognitive function using the Cambridge Neuropsychological Test Automated Battery (CANTAB)
    • Change from baseline in distance walked as measured by 6MWT
    • Time to onset of paroxysmal exertional dyskinesia (PED) as measured during 6MWT.
    • Change from baseline in gross motor function using the Gross Motor Function Measure-88 (GMFM-88)

    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 3 - 17 years (inclusive) at the time of informed consent
    3) Average of at least 5 observable seizures (generalized [except absence] or partial-onset [simple partial motor, complex partial, or secondarily generalized] seizures) per month over the last 6 months, by subject or caregiver report
    4) At least 4 observable seizures (generalized [except absence] or partial-onset [simple partial motor, complex partial, or secondarily generalized] seizures) per month during the Baseline Period, with no 3-week seizure-free period during the Baseline Period
    5) Continuing to have seizures despite a prior or current use of at least 1 AED
    6) Allowed to be on 1 - 3 concomitant AEDs that must have been stable in dose at least 2 weeks prior to the beginning of screening and anticipated to remain stable in dose through the end of the 8-week, placebo-controlled Treatment Period
    7) Not on, or not fully compliant with a prescribed diet plan (e.g. ketogenic diet) comprised of at least 50% total daily caloric intake from fat during previous 60 days (confirmed by 3-day diet diary at Screening), or at any time during the course of the trial
    8) Plasma level of beta-hydroxybutyrate (BHB) ≤ 1 mmol/L (non-fasting) at Screening
    9) Naïve to triheptanoin
    10) Provide written or verbal assent (if possible) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
    11) Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, comply with accurate completion of the seizures diary, and likely to complete the 8 week, placebo-controlled, Treatment Period
    12) Females who have reached menarche must have a negative pregnancy test at Screening. If sexually active, subject must be willing to use acceptable method of contraception and have additional pregnancy tests during the study.
    E.4Principal exclusion criteria
    1) Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels exceeding 2X the upper limit of normal at Screening
    2) Any known hypersensitivity to triheptanoin or safflower oil that, in the judgment of the investigator, places the subject at increased risk for adverse effects
    3) History of, or current suicidal ideation, behavior and/or attempts
    4) Breastfeeding an infant at Screening
    5) Participants unwilling or unable to discontinue use of a prohibited medication or other substance that may confound study objectives
    6) Use of any investigational product (drug or supplement, including medium chain triglyceride [MCT] oil) within 30 days prior to Screening, or at any time during the study
    7) Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment
    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 (e.g., diabetes mellitus, other concurrent neurological or psychiatric disorders)
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy evaluation is the reduction from baseline in monthly frequency of generalized or partial-onset seizures between Weeks 2 and 8. The baseline monthly frequency is defined as the number of generalized or partial-onset seizures experienced in the 6 weeks prior to randomization, normalized to a 4-week rate. The primary efficacy comparison, as well as secondary and exploratory analysis of the primary endpoint will be described in the SAP.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 8
    E.5.2Secondary end point(s)
    The secondary efficacy analyses are primarily focused on alternative domains of Glut1 DS disease to assess the breadth of treatment effect of UX007. The secondary analyses will compare the secondary efficacy variables scores after treatment to before treatment between the treated and placebo groups. The specific tests and analyses will be defined in the SAP.
    Included in these analyses will be a predefined per protocol analysis of those subjects that achieve at least 30% of calorie intake or higher as the tolerated dose level and have received at least 80% of the expected doses, to assess whether a subpopulation of the highest dose subjects have a larger difference in efficacy.
    Each of the efficacy variables assessed at Week 52 (or Week 26 for EEG only) will be compared to the value obtained at the baseline visit (or screening visit if not performed at the baseline visit) utilizing the same statistical test as conducted at the Week 8 analysis, but adjusted for the difference that the analyses for the Week 52 data will only utilize a within-cohort comparison and not a between-cohort comparison.
    Change-from-baseline values (where applicable) will be provided for the following secondary efficacy variables at baseline and Week 8:
    • The reduction from baseline to Week 8 in frequency of generalized tonic-clonic seizures.
    • Seizure response rate, defined as the percentage of subjects with at least 50% reduction from baseline in generalized or partial-onset seizures.
    • The change from baseline to Week 8 EEG abnormalities (including absence seizures) as determined by overnight EEG
    • The change from baseline to Week 8 in the individual scores for the CANTAB MOT, PAL, RTI, SSP and SWM tests.
    • The change from baseline to Week 8 6MWT distance and percent of predicted 6MWT distance.
    • Time to onset of PED during 6MWT.
    • The change from baseline to Week 8 in the GMFM-88 total score and 5 domain scores.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 52
    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 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 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 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 EEA4
    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
    France
    Italy
    Netherlands
    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 years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 80
    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: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 80
    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 8-week double-blind Treatment Period, the open-label Extension Period will begin, wherein all subjects will be treated with UX007 through Week 52 of the study. Beyond this period, subjects may be eligible for an extension study, if warranted, through a separate protocol.
    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 Decision2014-02-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-12-16
    P. End of Trial
    P.End of Trial StatusCompleted
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