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    Summary
    EudraCT Number:2013-004830-14
    Sponsor's Protocol Code Number:UX007-CL201
    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:2014-02-13
    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 number2013-004830-14
    A.3Full title of the trial
    An Open-Label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A safety and efficacy study in patients with Long-Chain Fatty Acid
    Oxidation Disorders receiving UX007 treatment
    A.4.1Sponsor's protocol code numberUX007-CL201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01886378
    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 pointDevon Jackson
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number14154838813
    B.5.6E-maildjackson@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/3/15/1508, EU/3/15/1524, EU/3/15/1525, EU/3/15/
    D.3 Description of the IMP
    D.3.1Product nametriheptanoin
    D.3.2Product code UX007
    D.3.4Pharmaceutical form Oral liquid
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
    E.1.1.1Medical condition in easily understood language
    Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    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
    Evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD (following 24 weeks treatment)
    E.2.2Secondary objectives of the trial
    • Evaluate the safety of UX007 treatment in subjects with LC-FAOD
    • Evaluate the effect of UX007 on energy metabolism in LC-FAOD
    • Evaluate the impact of UX007 on major clinical events associated with LC-FAOD (following 78 weeks treatment)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Confirmed diagnosis of one of the three most common LC-FAOD disorders: CPT II deficiency, VLCAD deficiency, LCHAD deficiency and TFP deficiency. Diagnosis must be confirmed by results of acylcarnitine profiles, fatty acid oxidation probe studies in cultured fibroblasts, and/or mutation analysis obtained from medical records.
    • Male or female, at least 6 months of age
    • Willing and able to complete all aspects of the study through the end of the study, including visits and tests, documentation of symptoms and diet, and administration of study medications. If a minor, have a caregiver(s) willing and able to assist in all applicable study requirements.
    • Provide written informed consent (subjects aged ≥ 18 years), or provide written assent (where appropriate) and have a legally authorized representative willing and able to provide written informed consent, after the nature of the study has been explained and prior to any research-related procedures
    • Willing and able to provide access to medical records charting the last 18-24 months of care prior to the study initiation, or from birth for those subjects less than 18 months of age
    • No history of serious adverse reactions or known hypersensitivity to triheptanoin
    • Currently managed on a stable treatment regimen (including diet), which may include low-fat/high carbohydrate diet, avoidance of fasting, carnitine and/or MCT oil. The treatment regimen (including diet) should be stable for the last 60 days to assure that changes in the subjects' condition are not confounded by recent changes in the treatment regimen that could affect the 4 week run-in evaluation period. Once
    study drug treatment has started, must be willing to maintain all aspects of the subject's treatment regimen and diet unchanged, other than discontinuation of MCT oil, in order to avoid potential variability of response due to variations in dietary intake.
    • Have severe LC-FAOD, as evidenced by ANY ONE of the following significant clinical manifestations despite therapy:
    - Chronic Elevated CK with Major Clinical Events: Elevated mean CK levels over the last 6 months -1 year (defined as ≥ 2X upper limit of age/gender-matched normal, or ≥ 500 units/L if age-matched reference not established) not associated with an acute rhabdomyolysis event, AND at least two major clinical events (as defined in the protocol) in the last year, or at least four major clinical events over the last two
    years,
    -Episodic Elevated CK with Reported Muscle Dysfunction: Episodes of elevated CK levels over the last 6 months -1 year (defined as ≥ 2X upper limit of age/gender-matched normal, or ≥ 500 units/L if age-matched reference is not established), AND patient report of frequent muscle fatigue, exercise intolerance, or limitation of exercise,
    - Highly Elevated CK but Asymptomatic: More seriously elevated mean CK levels (defined as ≥ 4X upper limit of age/gender-matched normal, or ≥ 1000 units/L if age-matched reference is not established) consistent with substantial chronic muscle rupture over the last 6 months-1 year,
    regardless of frequency hospitalizations or ER events,
    - Frequent Severe Major Medical Episodes (at least 3 within the past year, or 5 within 2 years) of hypoglycemia, rhabdomyolysis, or exacerbation of CM, requiring ER/acute care visits or hospitalizations,
    - Severe Susceptibility to Hypoglycemia (serum glucose <60 mg/dL) after short periods of fasting (less than 4-12 hours, depending on age), with at least 2 events in the last year that require ongoing prophylactic management, OR recurrent symptomatic hypoglycemia (blood glucose levels or clinical symptoms of hypoglycemia) at home requiring intervention ≥ 2 times per week,
    - Evidence of Functional CM (with echocardiogram (ECHO) within past 90 days documenting poor EF) requiring ongoing medical management
    • 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
    • Diagnosis of CACT deficiency, or CPT I
    • Diagnosis of medium-chain acyl-CoA dehydrogenase (MCAD) deficiency, short- or medium-chain FAOD, ketone body metabolism defect, propionic acidemia or methylmalonic acidemia
    • Enrolled in a clinical study involving concurrent use of an investigational drug product within the last 30 days, or unwilling to discontinue use of a prohibited medication or other substance that may confound study objectives
    • Unwilling to sign informed consent or release of medical records
    • Have any co-morbid conditions, including unstable major organ-system disease(s) that in the opinion of the Investigator, places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives
    E.5 End points
    E.5.1Primary end point(s)
    - Effects of UX007 on LC-FAOD will be assessed by evaluating critical variables of disease impacting three relevant disease areas (skeletal myopathy, hepatic disease, and cardiac disease), along with relevant biomarkers.
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the Treatment Period (24 weeks), three relevant disease areas will be assessed at Weeks 0, 4, 8, 12, 18, and 24, including 1) Skeletal myopathy: complications such as hypotonia, exercise intolerance, motor development, and functional disability, as well as non-acute CK levels (not associated with an acute rhabdomyolysis event), 2) Hepatic disease: hypoglycemia and related interventions and complications, including hepatomegaly and markers of hepatic function, and 3) Cardiac disease: ejection fraction, shortening fraction, and cardiac biomarkers.
    In addition, FAOD-related laboratory measures excluding CK levels will be assessed at Weeks 0, 4, 12 and 24. CK levels will be assessed at Weeks 0, 4, 8, 12, 18, and 24.
    E.5.2Secondary end point(s)
    - Safety events will be collected as adverse events (AE) or serious adverse events (SAE), including clinical laboratory tests, vital signs, physical examination, and concomitant medications.
    - The effects of UX007 treatment on major clinical events will be assessed over the Extension Period (total of 78 weeks). Major events are defined as musculoskeletal, hepatic, and cardiac events caused by LCFAOD, or intercurrent illness complicated by LC-FAOD, resulting in any hospitalization, ER visit, or emergency intervention (any unscheduled administration of therapeutics at home or in the clinic).
    - Evaluate the effect of UX007 on energy metabolism in LC-FAOD by measuring plasma UX007, UX007 metabolites.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Safety: Assessed up to Week 78. All AEs will be recorded from the time the subject signs the informed consent.
    - The effects of UX007 treatment on major clinical events will be assessed over the Extension Period (total of 78 weeks). Major events are defined as musculoskeletal, hepatic, and cardiac events caused by LC-FAOD, or intercurrent illness complicated by LC-FAOD, resulting in any hospitalization, ER visit, or emergency intervention (any unscheduled administration of therapeutics at home or in the clinic). Assessed at 4-week run-in period, Baseline, and Weeks 0, 4, 8, 12, 18, 24, 36, 48, 60, 78.
    - Evaluate the effect of UX007 on energy metabolism in LC-FAOD by measuring plasma UX007, UX007 metabolites, assessed at Weeks 0, 4, 12 and 24.
    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 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 No
    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 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 concerned3
    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 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
    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
    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 months9
    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 months9
    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: 20
    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.1.1.4.1Number 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: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 patients
    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 6
    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)
    Patients who complete all study requirements in Study UX007-CL201 may be eligible to participate in an extension study, if offered. However, patients are not obligated to participate in any additional studies, and the sponsor cannot guarantee that such studies will occur.
    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 Decision2014-02-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-02-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-08-25
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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