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    Summary
    EudraCT Number:2020-002100-39
    Sponsor's Protocol Code Number:PTC743-MIT-001-EP
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-12-14
    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 ConcernedSweden - MPA
    A.2EudraCT number2020-002100-39
    A.3Full title of the trial
    Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
    A.3.2Name or abbreviated title of the trial where available
    MIT-E
    A.4.1Sponsor's protocol code numberPTC743-MIT-001-EP
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04378075
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/318/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPTC THERAPEUTICS, 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 supportPTC THERAPEUTICS, INC.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPTC THERAPEUTICS, INC.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address100 CORPORATE COURT
    B.5.3.2Town/ citySOUTH PLAINFIELD
    B.5.3.3Post codeNJ 07080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 866-282-5873
    B.5.6E-mailmedinfo@ptcbio.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 nameVatiquinone
    D.3.2Product code PTC743
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNVATIQUINONE
    D.3.9.1CAS number 1213269-98-7
    D.3.9.2Current sponsor codePTC743
    D.3.9.3Other descriptive nameEPI743; alpha-tocotrienol quinone
    D.3.9.4EV Substance CodeSUB188275
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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
    Genetically determined mitochondrial disease and associated refractory epilepsy
    E.1.1.1Medical condition in easily understood language
    Mitochondrial disease associated with refractory epilepsy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate the effect of vatiquinone (PTC743) on reduction in observable motor seizure frequency in subjects with genetically confirmed mitochondrial disease, as assessed by a seizure diary.
    E.2.2Secondary objectives of the trial
    ● To demonstrate the effects of vatiquinone on seizure-related emergency room visits and hospitalizations
    ● To demonstrate the effects of vatiquinone on occurrence of status epilepticus
    ● To demonstrate the effects of vatiquinone in monthly total seizure count
    ● To demonstrate the effects of vatiquinone in responder rate in motor and non-motor seizures
    ● To demonstrate the effects of vatiquinone on number of rescue antiepileptic medications used
    ● To demonstrate the effects of vatiquinone on health-related quality of life (using the CarerQoL-7D questionnaire)
    ● To demonstrate the effects of vatiquinone on occurrence of seizure clusters
    ● To demonstrate the safety of vatiquinone as assessed by drug-related SAEs, drug-related adverse events (AEs), and dose modifications
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Signed informed consent form.
    • Age <21 years at time of randomization.
    •Subjects with genetic confirmation of inherited mitochondrial disease with associated epilepsy phenotype (Alpers/POLG, Leigh syndrome, MELAS), or other genetically confirmed mitochondrial diseases secondary to mitochondrial mutations (Pontocerebellar Hypoplasia Type 6 [PCH6], nuclear DNA RARS2 mutation) or myoclonic epilepsy with ragged red fibers (MERRF, mitochondrial DNA [mtDNA] mitochondrially encoded tRNA lysine [MT-TK] mutation) are eligible.

    •Despite treatment with at least 2 antiepileptic drugs:
    ◦have ≥6 observed motor seizures occurring during the 28 days prior to the baseline visit (Day 0).
    ◦have ≥2 observed motor seizures in the first 14 days and ≥2 in the second 14 days of the Run-in period (Day -14).
    ◦do not have a consecutive 20-day seizure free period.
    ◦have at least 80% of seizure diary data.
    No changes to the AED regimen will be allowed (except weight-based dose adjustments) during the first 24-week period.

    •Documented medical history of epilepsy symptoms associated with mitochondrial disease for at least 6 months prior to screening except for subjects who are <2 years of age at the time of screening (subjects <2 years of age can be considered for enrollment if all other screening criteria are met due to the potential for rapid progression in these subjects).
    •Consent to abstain from non-approved therapies for 30 days prior to the baseline visit (Day 0) and for the duration of the study.
    •Stable dose regimen of antiepileptic therapies 30 days prior to the screening visit
    •Stable regimen of dietary supplements 30 days prior and, if on a ketogenic diet, stable ketogenic diet 90 days prior to the screening visit and for duration of the study.
    •Electroencephalogram (EEG) at screening or historical EEG up to 6 months prior to screening for diagnostic confirmation of epilepsy.
    E.4Principal exclusion criteria
    •Allergy to vatiquinone or sesame oil.
    •Aspartate transaminase (AST) or alanine transaminase (ALT) ≥3×ULN at time of screening
    •INR > ULN at time of screening
    •Serum creatinine ≥1.5 × ULN at time of screening.
    •Participation in another interventional clinical trial 60 days prior to randomization or for the duration of this clinical trial
    •Previously received vatiquinone.
    •Concomitant treatment with drug(s) that have not received regulatory agency approval for the treatment of mitochondrial diseases and use of artisanal (non-Epidiolex cannabidiol) cannabidiol therapies.
    •Ongoing treatment with strong cytochrome P450 (CYP) inhibitors such as itraconazole or strong CYP inducers such as rifampin. Treatment with these agents must be completed at least 4 weeks prior to enrollment.
    •Pregnant or lactating participants or those male and female sexually active subjects who are unwilling to comply with proper birth control methods. Females of childbearing potential must have a negative pregnancy test at screening and during the baseline visit (Day 0).
    •Comorbidities that may confound study results (eg, fat malabsorption syndrome, other mitochondrial disorders) in the opinion of the investigator
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from Baseline to Week 24 in the Number of Observable Motor Seizures per 28 Days during the placebo-controlled phase
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 0, Week 24
    E.5.2Secondary end point(s)
    1.Number of Disease-Related Hospital Days
    2.Number of Participants with Occurrences or Recurrence of Status Epilepticus
    3.Number of Participants with Disease-Related In-Patient Hospitalizations or Emergency Room Visits
    4.Number of Disease-Related In-Patient Hospital Admissions or Emergency Room Visits
    5.Change from Baseline to Week 72 in Total Seizure Frequency (All Types) per 28 Days
    6.Percentage of Participants with ≥25%, ≥50%, ≥75%, and 100% Reduction in Motor Seizures
    7.Percentage of Participants with ≥25%, ≥50%, ≥75%, and 100% Reduction in Total Seizures (All Types)
    8.Number of Participants Who Require Rescue Seizure Medication
    9.Health-Related Quality of Life as Measured by the Care-Related Quality of Life of Informal Caregivers (CarerQoL-7D) Questionnaire
    10.Number of Participants with Seizure Clusters
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Week 24 and up to Week 72
    2. Week 24 and up to Week 72
    3. Week 24 and up to Week 72
    4. Week 24 and up to Week 72
    5. Day 0, Week 24, Week 72
    6. Week 24 and up to Week 72
    7. Week 24 and up to Week 72
    8. Week 24 and up to Week 72
    9. Week 24 and up to Week 72
    10. Week 24 and up to Week 72
    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) Yes
    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) 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 EEA12
    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
    Canada
    France
    Germany
    Italy
    Japan
    Poland
    Russian Federation
    Spain
    Sweden
    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 months8
    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 months7
    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: 88
    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) Yes
    F.1.2.1Number of subjects for this age range: 8
    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 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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 88
    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)
    None
    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 Decision2022-02-08
    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 Status
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