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    Summary
    EudraCT Number:2022-001539-10
    Sponsor's Protocol Code Number:UX007-CL302
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2023-01-20
    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 ConcernedGermany - BfArM
    A.2EudraCT number2022-001539-10
    A.3Full title of the trial
    A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared with Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
    A.4.1Sponsor's protocol code numberUX007-CL302
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/034/2023
    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 pointUX007-CL302 Clinical 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.5.6E-mailUX007-CL302@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 Yes
    D.2.1.1.1Trade name Dojolvi®
    D.2.1.1.2Name of the Marketing Authorisation holderUltragenyx Pharmaceutical Inc
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 (others listed in the Cover Letter)
    D.3 Description of the IMP
    D.3.1Product nameDojolvi®
    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 IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTriheptanoin
    D.3.9.1CAS number 620-67-7
    D.3.9.2Current sponsor codeUX007
    D.3.9.4EV Substance CodeSUB188284
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Miglyol® 812 N
    D.2.1.1.2Name of the Marketing Authorisation holderIOI Oleochemical
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameMiglyol® 812 N
    D.3.4Pharmaceutical form Oral liquid
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMedium-Chain Triglycerides
    D.3.9.3Other descriptive nameTRIGLYCERIDES, MEDIUM CHAIN
    D.3.9.4EV Substance CodeSUB12373MIG
    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 Yes
    D.3.11.13.1Other medicinal product typeDietary Supplement
    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] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077951
    E.1.2Term Fatty acid oxidation 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
    Evaluate the effect of triheptanoin versus MCT on frequency of MCEs
    E.2.2Secondary objectives of the trial
    - Evaluate the effect of triheptanoin versus MCT on reducing the duration of MCEs
    - Evaluate the effect of triheptanoin versus MCT on reducing the frequency of hypoglycemic events captured as MCEs and/or HCEs
    - Evaluate the effect of triheptanoin versus MCT on clinician-reported change in overall health status and functioning
    - Evaluate the effect of triheptanoin versus MCT on reducing the frequency of cardiomyopathy MCEs
    - Evaluate the effect of triheptanoin versus MCT on hepatic lipids (Liver Substudy – Primary)
    - Evaluate the effect of triheptanoin versus MCT on echocardiographic assessments
    - Assess the contribution of each component MCE (rhabdomyolysis, cardiomyopathy, and hypoglycemia) to the composite MCE primary endpoint (which includes all 3 types of MCEs)
    - Assess the effect of triheptanoin versus MCT on physical health, psychosocial health, and total HRQoL
    - Evaluate the effect of triheptanoin versus MCT on all-cause mortality
    Add. sec. object. in protocoll
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    A Liver sub-study will be conducted at selected sites with appropriate expertise, facilities, and equipment.

    (...) details about this sub-study can be found in the protocol (section 7.1.2)
    E.3Principal inclusion criteria
    1. Confirmed diagnosis of LC-FAOD: carnitine palmitoyl transferase (CPT) I deficiency, CPT II deficiency, carnitine/acylcarnitine translocase (CACT) deficiency, very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxyacyl-CoA
    dehydrogenase (LCHAD) deficiency, and mitochondrial trifunctional protein (TFP) deficiency. Diagnosis must be confirmed by results of acylcarnitine profiles, fatty acid oxidation probe studies in cultured fibroblasts, or mutation analysis obtained from medical records

    2. Males and females, from newborn to < 17 years of age

    3. Have a caregiver(s) willing and able to assist in all applicable study requirements

    4. 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, and the study subject to be able to provide age-appropriate written assent

    5. Have ANY ONE of the following significant clinical manifestations of LC-FAOD:
    - At least 2 in the prior year, or 3 in the prior 2 years, of severe major episodes of metabolic decompensation (eg, hypoglycemia, rhabdomyolysis, or exacerbation of cardiomyopathy, requiring ER/urgent care unit visits or hospitalizations)
    - Recurrent symptomatic hypoglycemia (clinical symptoms of hypoglycemia) requiring intervention
    - Susceptibility to hypoglycemia after short periods of fasting (less than 4 to 12 hours, depending on age)
    - Evidence of functional cardiomyopathy requiring ongoing medical management or clinical manifestation of heart failure
    - Sibling(s) with the same pathogenic variant who presented with MCEs
    - Subject with pathogenic variants that are known or suspected to be associated with absent or severely reduced enzyme activity or with severe disease manifestations.

    6. From the time of informed consent to 5 days after the last dose of study drug in this study, females of childbearing potential and fertile males must consent to use highly effective methods of contraception as described in Appendix 2. If female, agree not to become pregnant. If male, agree not father a child or donate sperm.

    Inclusion Criteria for Liver Sub-study
    1. Enrollment in the Main Study of Study UX007-CL302

    2. Age > 2 years

    3. Liver fat content ≥ 2% and < 20% PDFF as assessed by 1H-MRS

    4. Body mass index < 95th percentile

    5. Able to comply with instructions (remaining still during scan) and requirements (eg, constraints on recent meals, no metallic items or implanted devices in the body, no recent contrast agents) for liver H-MRS scan
    E.4Principal exclusion criteria
    1. Enrolled in a clinical study involving concurrent use of an investigational drug product within 30 days before Screening

    2. Use of a prohibited medication (eg, valproate products or pancreatic lipase inhibitors) within 30 days before Screening, or unwilling to avoid a prohibited medication or other substance that may confound study objectives

    3. Treatment with triheptanoin within 60 days of Screening

    4. History of known hypersensitivity to triheptanoin or MCT

    5. Caregiver unwilling or unable to sign informed consent, or release of medical records, or follow study procedures

    6. 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. History of metabolic decompensation(s) with metabolic acidosis, hyperammonemia, and/or liver enzyme elevations does not constitute an exclusion criterion unless in the opinion of the Investigator places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives.

    7. Have a diagnosis of pancreatic insufficiency

    8. Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study.

    Exclusion Criteria for Liver Sub-study
    1. Acute or chronic liver disease other than LC-FAOD that presents with increased risk of liver fat (eg, hepatic cirrhosis, viral toxic or drug hepatitis, diabetes mellitus) and/or metabolic syndrome
    2. Need for anesthesia/sedation to perform liver H-MRS
    E.5 End points
    E.5.1Primary end point(s)
    Annualized event rate of MCEs during the Double-blind Treatment Period
    E.5.1.1Timepoint(s) of evaluation of this end point
    Annually
    E.5.2Secondary end point(s)
    - Annualized duration of MCEs during the Double-blind
    Treatment Period
    - Annualized hypoglycemic event-rate captured as MCEs and/or HCEs during the Double-blind Treatment Period
    - Clinician-reported change in global impression of
    disease severity (Clinical Global Impression of Change [CGI-C] scale) score at EOS
    - Annualized frequency of cardiomyopathy-MCEs during the Double-blind Treatment Period
    - Left ventricular ejection fraction, left ventricular systolic volume, and
    left ventricular wall mass, if available
    - Change from baseline to 6 months in hepatic PDFF%,
    assessed by 1H-MRS in subjects enrolled in the Liver Substudy
    - Annualized frequency and duration of rhabdomyolysis-MCEs during the Double-blind Treatment Period
    - Annualized frequency and duration of cardiomyopathy-MCEs during the Double-blind Treatment Period
    - Annualized duration of hypoglycemic-MCEs during the Double-blind Treatment Period
    - Change from baseline to EOS in scores for: Caregiver-reported PedsQL 4.0 Generic Core Scale (physical health summary, psychosocial health summary, and total scores) (2 years of age
    and older) OR PedsQL Infant Scale (physical health summary,
    psychosocial health summary, and total scores) (ages 1 to < 24 months)
    - Survival time during the Double-blind Treatment Period
    - Change from baseline to 6 months in CK/ALT, ALT, AST in subjects enrolled in the Liver Substudy
    - Annualized hospitalization days during the Double-blind Treatment Period
    - Number of missed school or learning opportunity days during the Double-blind Treatment Period
    - Frequency, severity, and relationship to study drug of TEAEs, serious TEAEs, and AESIs
    - Incidence of TEAEs and serious TEAEs leading to dose modifications, dose reductions, treatment interruptions, discontinuations from study drug, and discontinuations from the study
    - Plasma concentration levels of heptanoate and betahydroxypentanoate (BHP)
    - Acceptability and Palatability Survey scores of triheptanoin mixed with oral liquids
    E.5.2.1Timepoint(s) of evaluation of this end point
    N/A - variable
    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 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 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) Yes
    E.8.2.2Placebo No
    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 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
    Czechia
    Germany
    Poland
    Saudi Arabia
    Spain
    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
    The end of the study is defined as when the last subject enrolled has completed 2 years of treatment.
    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 days
    E.8.9.2In all countries concerned by the trial years4
    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: 60
    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: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 22
    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 No
    F.3.2Patients No
    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.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children from newborn to < 18 years of age
    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 25
    F.4.2.2In the whole clinical trial 60
    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)
    Subjects who complete or withdraw from the study may have access to continued treatment under company-sponsored compassionate use policies depending on country regulations and drug availability. After drug approval subjects may be invited to participate in an LC-FAOD Disease Monitoring Program (DMP).
    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 Decision2023-03-21
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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