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    Summary
    EudraCT Number:2018-001846-32
    Sponsor's Protocol Code Number:BUS2018-1
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-07-31
    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 number2018-001846-32
    A.3Full title of the trial
    Treatment of NF1-related plexiform neurofibroma with trametinib; a single arm, open-label trial with the goals of volumetric partial remission and pain relief
    Behandling av NF1-relaterade plexiforma neurofibrom med trametinib; en enarmad, öppen studie med effektmått volumetrisk partiell remission och smärtlindring
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of symptomatic plexiform neurofibromas, a benign tumour associated with the disorder Neurofibromatosis type 1, in children with the drug trametinib
    Behandling av besvärliga plexiforma Neurofibrom, en tumör som
    förekommer vid sjukdomen Neurofibromatos typ 1, hos barn med läkemedelssubstansen trametinib
    A.3.2Name or abbreviated title of the trial where available
    plexifpc
    plexifpc
    A.4.1Sponsor's protocol code numberBUS2018-1
    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 SponsorVO Barnmedicin, Skånes University Hospital
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovartis Sverige AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVO barnmedicin, Skånes University Hospital, Lund
    B.5.2Functional name of contact pointAVD 63 / Björn Sigurdsson
    B.5.3 Address:
    B.5.3.1Street AddressGetingevägen 4
    B.5.3.2Town/ cityLund
    B.5.3.3Post code22241
    B.5.3.4CountrySweden
    B.5.4Telephone number0046708253552
    B.5.6E-mailbjorn.sigurdsson@skane.se
    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 Mekinist
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameMekinist
    D.3.4Pharmaceutical form Oral drops
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    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
    NF1-related plexiform neurofibroma
    NF1-relaterade plexiforma neurofibrom
    E.1.1.1Medical condition in easily understood language
    Plexiform Neurofibroma (PN). It is a tumour associated with
    neurofibromatosis type 1 (NF1).
    Plexiforma neurofibrom (PN). Det är en tumör hos personer med neurofibromatos typ 1 (NF1).
    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
    Remission of NF1-related plexiform neurofibroma (PN)
    E.2.2Secondary objectives of the trial
    Reversal of clinical symptoms of NF1-related PN
    Exploratory: Evaluate changes in cognitive performance
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    NF1-related PN with severe - or with high suspicion of threatening -
    manifestations
    • Informed consent given
    • Age 1:0-17:11
    E.4Principal exclusion criteria
    • NF1-related PN does not fulfill characteristics for acceptable volumetric MRI assessments as outlined in box Criterion for volumetric assessment.
    • Lactating or pregnant or sexual active females, who do not use safe contraception. Sexual active males who do not use condom.
    • History of other malignancies than classic NF1-related WHO grade 1 tumor (i.e. PN or optic pathway glioma).
    • Subjects with a history of NF-1 related cerebral vascular anomaly (such as Moyamoya).
    • Subjects with NF-1, receiving pharmaceutical therapy for optic pathway malignancy/ies.
    • Any medication for treatment of left ventricular systolic dysfunction.
    • Administration of an investigational study treatment within 30 days preceding the first dose of the study treatment in this study.
    • Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to the study treatment or excipients that contraindicate their participation.
    • Current active liver or biliary disease
    • History of hepatic sinusoid obstructive syndrome (venoocculsive disease) within the prior 3 months.
    • History of heparin-induced thrombocytopenia.
    • History of interstitial lung disease or pneumonitis.
    • History of or current evidence of retinal vein occlusion (RVO).
    • A history of known Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV) infection. Subjects with laboratory evidence of cleared HBV and HCV infection may be enrolled.
    • Presence of condition that will interfere significantly with the absorption of drugs.
    • Evidence of cardiovascular risk, LVEF below LLN, Qtc >480 millisecond, clinical significant uncontrolled arrhythmia, congestive heart failure, acute coronary syndrome or history thereof.
    E.5 End points
    E.5.1Primary end point(s)
    Remission of tumor volume ≥20% evaluated with volumetric MRI
    E.5.1.1Timepoint(s) of evaluation of this end point
    month 18 (interim analysis) and month final analysis at month 30.
    E.5.2Secondary end point(s)
    Reversal of clinical symptoms of NF1-related PN. Evaluated with pain diary, VAS scale or Faces Pain Scale, from month 0 to 30 following. Descriptive.

    Exploratory: Change of either; full scale IQ or primary indexes (WISC V), Learning
    and Memory functions and visuospatial functions (selected test from
    NEPSY II), or attention (Conners CPT3); with p-value <0.05
    E.5.2.1Timepoint(s) of evaluation of this end point
    month 18 (interim analysis) and month final analysis at month 30 for
    pain and after month 18 for cognitive function.
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 150
    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: 16
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 75
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 50
    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 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 No
    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
    Some children will be too young to be giving informed consent personally. In such case guardian(s) have to to be capable of giving informed consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    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 Decision2018-09-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-14
    P. End of Trial
    P.End of Trial StatusOngoing
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