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    Summary
    EudraCT Number:2011-004369-34
    Sponsor's Protocol Code Number:UMC-NMZ-SMA2011
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-11-03
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2011-004369-34
    A.3Full title of the trial
    SPACE trial
    SMA and Pyridostigmine in Adults and Children; Efficacy trial
    Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4
    SPACE trial
    SMA en Pyridostigmine in volwassenen en kinderen; doeltreffendheid trial
    Een fase II, mono-centrische, placebogecontroleerde cross-over trial naar effect en doeltreffendheid van pyridostigmine in volwassen Nederlandse patienten met spinale spieratrofie type 2, 3 en 4.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    SPACE trial
    SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4
    SPACE trial
    SMA en Pyridostigmine in volwassenen en kinderen; experimentele trial naar effect van pyridostigmine vergeleken placebo in volwassen Nederlandse patienten met spinale spieratrofie type 2, 3 en 4.
    A.3.2Name or abbreviated title of the trial where available
    SPACE trial
    SPACE trial
    A.4.1Sponsor's protocol code numberUMC-NMZ-SMA2011
    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 SponsorUniverstiy Medical Center Utrecht
    B.1.3.4CountryNetherlands
    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 supportPrinses Beatrix Fonds
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUMC Utrecht
    B.5.2Functional name of contact pointMETC
    B.5.3 Address:
    B.5.3.1Street AddressPostbus 85500
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3508 GA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number0031887556376
    B.5.6E-mailmetc@umcutrecht.nl
    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 pyridostigmine bromide
    D.2.1.1.2Name of the Marketing Authorisation holderCBG
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameMestinon
    D.3.2Product code RVG 03820
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    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
    Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive.
    Spinale spieratrofie (SMA) is een aandoening gekenmerkt door proximale spierzwakte door degeneratie van motorneuronen in de voorhoorncellen van het ruggenmerg. SMA wordt veroorzaakt door een homzygote deletie van het SMN1-gen. Er zijn 4 types SMA, geclassificeerd op basis van behaalde motorische mijlpalen en leeftijd van eerste symtpomen. De behandeling van SMA is ondersteunend.
    E.1.1.1Medical condition in easily understood language
    Spinal muscular atrophy (SMA) is a disorder characterized by muscle weakness caused by loss of motor cells of the spinal cord. SMA is a genentic disease. Treatment of SMA is supportive.
    Spinale spieratrofie (SMA) is een aandoening met proximale spierzwakte door verlies van motorische cellen in het ruggenmerg. SMA is een genetische aandoening. De behandeling van SMA is ondersteunend.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10068209
    E.1.2Term Spinal muscular atrophy adult onset
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10051203
    E.1.2Term Spinal muscular atrophy congenital
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of this placebo-controlled cross-over trial in adult patients with SMA type 2, 3 and 4 is to investigate the effect and efficacy of pyridostigmine on muscle strength and fatiquabillity in patients with SMA.
    Het hoofddoel van deze placebo-controlled cross-over trial bij volwassen patienten met SMA type 2, 3 en 4 is het onderzoeken van effect en doeltreffendheid van pyridostigmine op de spierkracht en het uithoudingsvermogen bij patiënten met SMA.
    E.2.2Secondary objectives of the trial
    Secondary outcome measures are the effect of pyridostigmine on neurofysiologic parameters and subjective scales
    Secundaire doelen van deze studies zijn het effect van pyridostigmine op neurofysiologische parameters en subjectieve scorelijsten.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients with SMA will be included following the predefined criteria: 1) a clinical diagnosis of SMA type 2, 3a, 3b or 4 and a genetically confirmed homozygous SMN1 deletion 2) ability to complete visits during trial period; 3) given oral and written informed consent when ≥18 years old; 4) given informed consent by the parents or legal representative in case of patients aged <18 years old; 5) ability of performing the Nine Hole Peg test within 50 seconds
    1) diagnose SMA type 2, 3a, 3b of 4 op basis van het klinisch beeld en bevestigd met een homozygote deletie van het SMN1-gen; 2) mogelijkheid tot het afleggen van alle visits en procedures gedurende de trialperiode; 3) schriftelijk en mondeling informed consent ≥18 jaar leeftijd; 4) schriftelijk en mondeling informed consent door ouders en/of voogd bij <18 jaar leeftijd; 5) Nine Hole Peg test binnen 50 seconden
    E.4Principal exclusion criteria
    Exclusion criteria for patients are 1) known concomitant disorders of the NMJ (Lambert Eaton myasthenic syndrome, myasthenia gravis); 2) use of drugs that may alter NMJ function; 3) SMA type 1; 4) apprehension against participation in EMG; 5) inability to meet study visits; 6) mechanical gastro-intestinal, urinary or biliary obstruction; 6) clinical significant alterations of laboratory tests (electrolytes, liver function, kidney function, thyroid function or blood dysplasia) drawn within 14 days prior to start of study entry; 7) electrocardiofysiology abnormalities known as a contraindication for pyridostigmine use; 8) pregnancy 9) allergy to bromides 10) bronchial astma
    1) bewezen aandoening van de neuromusculaire overgang (lambert eaton myastheen syndroom, myasthenia gravis) naast SMA; 2) gebruik van medicatie die de neuromusculaire overgang beïnvloeden; 3) SMA type 1; 4) weerstand tegen het ondergaan van repetitieve zenuwstimualtie; 5) onvermogen tot afleggen van alle visits; 6) mechanicshe obstructie gastro-intestinaal, urogenitaal of galwegen; 7) klinisch significante laboratoriumveranderingen (elektrolyten, leverfunctie, nierfunctie, schildklierfucntie of bloedbeeld); 7) afwijkend elektrocardiogram; 8) zwangerschap; 9) allergie voor bromiden; 10) bronchiale astma
    E.5 End points
    E.5.1Primary end point(s)
    Change in Nine Hole Peg test and Motor Function Measure
    Verandering in Nine Hole Peg test and Motor Function Measure
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patients will be screened. Clinical evaluation in patients will take place 2 times in de treatmentperiod of 8 weeks; at te start and at the end.
    Patienten worden gescreend. Tijdens de 8weekse- behandelperiode worden patienten op twee tijdsstippen ge-evalueerd, aan het begin (week1) en aan het eind (week 8)
    E.5.2Secondary end point(s)
    Secondary outcome measures are scores on Vigori Measurement, scales on daily activities and presence of decrement upon repetitive nerve stimulation, quality of and activity in life scores, and adverse events.
    Secundaire uitkomsten zijn veranderingen in de uitkomst van de Vigori Meter, MRC sumscore, scorelijsten over dagelijkse activiteiten, de aanwezigheid en verandering in decrementie bij repetitieve zenuwstimulatie en bijwerkingen.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Clinical evaluation in patients will take place 2 times in de treatmentperiod of 8 weeks; at te start and at the end.
    Tijdens de 8weekse- behandelperiode worden patienten op twee tijdsstippen ge-evalueerd, aan het begin (week1) en aan het eind (week 8)
    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 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 Yes
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 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 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 No
    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
    Last visit of last subject undergoing the trial
    Laatste bezoek van laatste participerende patient
    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 months6
    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: 12
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state45
    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)
    Patient can still use pyridostigmien if adequate follow-up by their doctor or the investigator is provided
    Patienten kunnen pyridostigmine na de trial blijven gebruiken wanneer adequate follow-up door hun behandelend arts of de onderzoeker wordt gegarandeerd.
    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 Decision2014-11-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-04-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-01-17
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