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    The EU Clinical Trials Register currently displays   41201   clinical trials with a EudraCT protocol, of which   6744   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
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    Summary
    EudraCT Number:2019-001147-51
    Sponsor's Protocol Code Number:COMPIS
    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:2019-09-27
    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 number2019-001147-51
    A.3Full title of the trial
    Congenital myopathy intervention study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a treatment study for a muscle disorder known as congenital myopathy
    A.3.2Name or abbreviated title of the trial where available
    COMPIS
    A.4.1Sponsor's protocol code numberCOMPIS
    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 SponsorVästra götalandsregionen
    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 supportVästragötalandsregionen
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVästra götalandsregionen
    B.5.2Functional name of contact pointBarnneurologen, DSBUS
    B.5.3 Address:
    B.5.3.1Street AddressRöndvägen 10
    B.5.3.2Town/ cityGothenburg
    B.5.3.3Post code41650
    B.5.3.4CountrySweden
    B.5.4Telephone number0046762883668
    B.5.6E-maileva.michael@vgregion.se
    D. IMP Identification
    D.IMP: 1
    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 Ventoline tablet 2mg
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxosmithkline
    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.3.4Pharmaceutical form Tablet
    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 INNSALBUTAMOL SULFATE
    D.3.9.1CAS number 51022-70-9
    D.3.9.4EV Substance CodeSUB04303MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number6 to 12
    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 Ventoline oral solution 0,4mg/ml
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxosmithkline
    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.3.4Pharmaceutical 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 INNSALBUTAMOL SULFATE
    D.3.9.1CAS number 51022-70-9
    D.3.9.4EV Substance CodeSUB04303MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number6 to 12
    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
    Congenital myopathy
    E.1.1.1Medical condition in easily understood language
    Congenital myopathy is a muscle disorder that presents with low muscle tone and generalised hypotonia. Symptoms present during childhood but in severe cases they appear from birth
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10062547
    E.1.2Term Congenital myopathy
    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
    We want to investigate if daily intake of oral salbutamol can increase the
    total score of muscle function measure 32 test in a group of congenital
    myopathy subjects after 6 months on treatment as compared to having
    no treatment.
    E.2.2Secondary objectives of the trial
    1) Increased walking distance during 6-minute walking test after 6 months of treatment
    2) Less fatigability during physical activity after 6 months of treatment: assessed by comparing subjects speed (m/min) during the first and last minute of 6 minute walking test
    3) Decreased time needed to complete timed function tests (10 m walk/run, 4 steps and down) after 6 months treatment
    4) Increased hand strength on hand held myometry after 6 months on treatment.
    5) Less fatigability in upper extremities during physical examination: assessed with 5 consecutive 9 hole peg tests, where time of completion of first set is compared with time of completion of the last set.
    6) Increase in forced vital capacity both sitting and lying down after 6 months of treatment
    7) Improvement of quality of life after 6 months of treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a) Signed informed consent from legal guardians/patients and patient (where applicable)
    b) Subject must have a confirmed CM diagnosis defined as:
    • Clinical symptoms consistent with CM with pathohistological findings on muscle biopsy and known genetic mutation consistent with CM
    OR
    • Clinical symptoms consistent with CM with unspecific pathohistological changes but known genetic mutation consistent with CM
    c) Stabile motor function tests over at least 6 months ( between baseline and screening visit.
    d) At least 1 point on MFM32 t screening visit
    e) If on other medications- stabile dose for at least 6 months prior to start
    f) >5- <31 years of age
    g) Women of fertile age must be on oral contraceptives
    h) Subjects must have had cardiac evaluation with ECG and 2D echocardiography in the last 2 years, with no signs or symptoms of cardiac abnormality.
    E.4Principal exclusion criteria
    a) Subject with clinical symptoms consistent with congenital myopathy but has no confirmed genetic mutation and only unspecific changes on muscle biopsy that are not confined to just congenital myopathy but can be seen in other disorders.
    b) Younger than 5 years of age and older than 31 years.
    c) Subject receives 94 or more points on MFM32 test at screening visit.
    d) Subject doesn’t not speak Swedish and a translator is needed in order to perform the tests included in the study.
    e) Subject receives no point on MFM 32 test at screening (baseline 2)
    f) Subject smokes more than 10 cigarettes a day or has smoked more that 10 cigarettes in the last year before the study start
    g) Subject has tracheostomy
    h) Subject has other concomitant chronic diagnosis that can affect the patients motor function, in the opinion of the investigator
    i) Subject is currently or has been on oral corticosteroids in the last 6 months
    j) Subject has arrhythmia as seen on ECG, confirmed by cardiologist
    k) Subject has cardiomyopathy as seen on ultrasound, confirmed by cardiologist
    l) Subject has severe behavioral and/ cognitive problems that preclude participation in the study, in the opinion of the investigator;
    m) Subject is allergic or hypersensitive to study drug or any of its constituents
    n) Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
    o) Subject is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study medication.
    p) Subject is planning on participating in any other study during the duration of this study.
    q) Female subjects of fertile age that are pregnant or are planning to become pregnant during the study.
    r) Female subjects that have given birth upto 1 year prior to first baseline visit and/or are nursing upto 1 month prior to first baseline visit.
    s) Subject has a fracture in the last 6 months before the study start or has acquired a fracture during the study.
    E.5 End points
    E.5.1Primary end point(s)
    Increase of MFM32 test with at least 3 points after 6 months of treatment as compared to no treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    -6months, 0 (randomization, study start), 6 months, 7 months and 13
    months after randomization
    E.
    E.5.2Secondary end point(s)
    1) Increased walking distance during 6-minute walking test after 6 months of treatment
    2) Less fatigability during physical activity after 6 months of treatment: assessed by comparing subjects speed (m/min) during the first and last minute of 6 minute walking test
    3) Decreased time needed to complete timed function tests (10 m walk/run, 4 steps and down) after 6 months treatment
    4) Increased hand strength on hand held myometry after 6 months on treatment.
    5) Less fatigability in upper extremities during physical examination: assessed with 5 consecutive 9 hole peg tests, where time of completion of first set is compared with time of completion of the last set.
    6) Increase in forced vital capacity both sitting and lying down after 6 months of treatment
    7) Improvement of quality of life after 6 months of treatment
    E.5.2.1Timepoint(s) of evaluation of this end point
    -6months, 0 (randomization, study start), 6 months, 7 months and 13
    months after randomization
    E.
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    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 Yes
    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 Yes
    E.8.2.3.1Comparator description
    no treatment
    E.8.2.4Number of treatment arms in the trial4
    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 years3
    E.8.9.1In the Member State concerned months8
    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: 15
    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: 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: 5
    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 state20
    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)
    The subjects will be followed-up up to a year after the study finishes. Then all the subjects will continue with their ordinary yearly follow-ups at their nearest university hospital
    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 Decision2019-11-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-31
    P. End of Trial
    P.End of Trial StatusOngoing
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