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    Summary
    EudraCT Number:2019-005007-40
    Sponsor's Protocol Code Number:S62874
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-01-16
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2019-005007-40
    A.3Full title of the trial
    Natural history study in adult patients with SMA types 2-3-4 and Role of neurodegenerative and neuro-inflammatory biomarkers in SMA adults treated with nusinersen.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Natural history study in adult patients with SMA types 2-3-4 and Role of neurodegenerative and neuro-inflammatory biomarkers in SMA adults treated with nusinersen.
    Studie van het natuurlijk ziekteverloop bij volwassen patiënten met spinale spieratrofie (SMA) en de rol van biomerkers bij SMA patiënten die met nusinersen behandeld worden.
    A.3.2Name or abbreviated title of the trial where available
    Adult-SMA
    A.4.1Sponsor's protocol code numberS62874
    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 SponsorUniversity Hospitals Leuven
    B.1.3.4CountryBelgium
    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 supportBiogen
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Hospitals Leuven
    B.5.2Functional name of contact pointDepartment of Neurology
    B.5.3 Address:
    B.5.3.1Street AddressHerestraat 49
    B.5.3.2Town/ cityLeuven
    B.5.3.3Post code3000
    B.5.3.4CountryBelgium
    B.5.4Telephone number3216344280
    B.5.5Fax number3216344285
    B.5.6E-mailsecretariaat.neuro@uzleuven.be
    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 Spinraza
    D.2.1.1.2Name of the Marketing Authorisation holderBiogen Netherlands B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/12/976
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 typeNusinersen is classified as an antisense oligonucleotide, a class of synthetic single stranded molecules of nucleic acids.
    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
    Adult patients with spinal muscular atrophy (SMA) type 2, type 3, or type 4
    E.1.1.1Medical condition in easily understood language
    spinal muscular atrophy (SMA)
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10041582
    E.1.2Term Spinal muscular atrophy
    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
    To perform a detailed clinical and functional characterization and natural history analysis in adult patients with SMA types 2-3-4 with and without nusinersen (Spinraza®) treatment.
    E.2.2Secondary objectives of the trial
    To assess effectiveness of nusinersen treatment in adult SMA patients. To assess safety of nusinersen treatment in adult SMA patients.
    To assess natural history in untreated adult SMA patients.
    To identify biomarkers in blood and cerebrospinal fluid correlated to clinical outcomes in adult SMA patients treated with nusinersen.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    (1) Male or female patients, adult age (18 years or older), with genetically confirmed 5q-related Spinal Muscular Atrophy (SMA) (i.e. deletions of both SMN1-genes or mutations in both SMN1- genes have been proven).
    (2) Female patients need to use a contraceptive in case they are being treated with nusinersen (Spinraza®) (standard-of-care).
    (3) Written informed consent.
    E.4Principal exclusion criteria
    (1) Patients younger than 18 years of age at the time of the study.
    (2) Patients without genetically confirmed diagnosis of 5q-SMA at the time of the study.
    (3) No written informed consent.
    E.5 End points
    E.5.1Primary end point(s)
    Change in clinical and functional characteristics (*) in adult SMA patients treated with nusinersen from baseline to 22 months.

    (*) The clinical and functional characteristics are the following: 6 minute walk distance, grip strength at both hands, general clinical neurological examination, Medical Research Council (MRC) sum score, Hammersmith functional motor scale expanded (HFMSE) with video-acquisition, revised upper limb module (RULM) with video-acquisition, lung function testing in sitting and supine position, the Rasch-built measure of activity limitations scale (ActiVlim), RX full-spine, blood analyses, urine analysis, vital parameters such as blood pressure, pulse, temperature, and the SF-36 quality of life (QoL) questionnaire.
    E.5.1.1Timepoint(s) of evaluation of this end point
    22 months after start of treatment
    E.5.2Secondary end point(s)
    Number of adult SMA patients that experience adverse events of nusinersen treatment.
    Decline in clinical and functional measurements in adult SMA patients that have not been treated.
    Quantitative changes of biomarkers in blood and CSF over time during treatment with nusinersen in adult SMA patients
    E.5.2.1Timepoint(s) of evaluation of this end point
    22 months after start of treatment
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 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
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state19
    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 treatment with nusinersen (Spinraza®) is standard-of-care treatment in patients with SMA and will thus be continued after the trial has ended
    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 Decision2020-02-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-08-30
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