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    Summary
    EudraCT Number:2020-003492-18
    Sponsor's Protocol Code Number:232SM404
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-08
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-003492-18
    A.3Full title of the trial
    A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
    Studio di fase 4 su nusinersen (BIIB058) in pazienti con atrofia muscolare spinale che hanno ricevuto onasemnogene abeparvovec
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
    Uno studio su Nusinersen tra i partecipanti con atrofia muscolare spinale che hanno ricevuto Onasemnogene Abeparvovec
    A.3.2Name or abbreviated title of the trial where available
    Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
    Studio su nusinersen (BIIB058) in partecipanticon atrofia muscolare spinale
    A.4.1Sponsor's protocol code number232SM404
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04488133
    A.5.4Other Identifiers
    Name:INDNumber:110011
    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 SponsorBIOGEN IDEC RESEARCH LIMITED
    B.1.3.4CountryUnited Kingdom
    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 supportBiogen Idec Research Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiogen Idec Research Limited
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street AddressInnovation House, 70 Norden Road
    B.5.3.2Town/ cityMaidenhead
    B.5.3.3Post codeSL6 4AY
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailclinicaltrials@biogen.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 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 AuthorisationNetherlands
    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.3.1Product name.
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNusinersen
    D.3.9.1CAS number 125894-36-9
    D.3.9.2Current sponsor codeISIS 396443 (BIIB058)
    D.3.9.3Other descriptive nameNUSINERSEN SODIUM
    D.3.9.4EV Substance CodeSUB189898
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2400
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 type2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide
    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
    Muscular Atrophy, Spinal
    atrofia muscolare spinale
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy (SMA)
    atrofia muscolare spinale (SMA)
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    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
    The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec.
    L'Obiettivo primario di questo studio è di valutare gli esiti clinici dopo il trattamento con nusinersen in partecipanti con AMS che hanno ricevuto in precedenza onasemnogene abeparvovec
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the safety and tolerability; and clinical outcomes following treatment with nusinersen in participants with SMA who previously received onasemnogene abeparvovec.
    Gli obiettivi secondari di questo studiosono di Valutare la sicurezza e la tollerabilità, gli esiti clinici dopo il trattamento con nusinersen nei partecipanti con AMS che hanno ricevuto in precedenza onasemnogene abeparvovec
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).
    - Must have previously received onasemnogene abeparvovec.
    - Participants with suboptimal clinical status per the Investigator.
    - Documentazione genetica della SMA 5q (delezione gene in omozigosi, mutazione o eterozigote composto)
    - Devono aver precedentemente ricevuto onasemnogene abeparvovec.
    - Partecipanti con stato clinico subottimale secondo lo sperimentatore.
    E.4Principal exclusion criteria
    - Prior exposure to nusinersen.
    - Ongoing severe or serious AEs related to onasemnogene abeparvovec.
    - Use of steroids within 1 month prior to first dose of nusinersen.
    - Precedente esposizione a nusinersen.
    - EA gravi o seri in corso correlati a onasemnogene abeparvovec.
    - Uso di steroidi entro 1 mese prima della prima dose di nusinersen.
    E.5 End points
    E.5.1Primary end point(s)
    Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score
    Punteggio totale nelle tappe di sviluppo motorio dell’esame neurologico del neonato di Hammersmith (HINE), sezione 2
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to Day 778
    Fino al Giorno 778
    E.5.2Secondary end point(s)
    1) Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
    2) Number of Participants with Change from Baseline in Clinical Laboratory Parameters
    3) Number of Participants with Change from Baseline in Electrocardiograms (ECGs)
    4) Number of Participants with Change from Baseline in Vital Signs
    5) Number of Participants who Achieved Motor Milestones as Assessed by World Health Organization (WHO) Criteria
    6) Change from Baseline in Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score
    7) Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
    8) Change from Baseline in Revised Upper Limb Module (RULM) Score
    9) Time to Death or Permanent Ventilation
    1) Numero di partecipanti con eventi avversi (EA) ed eventi avversi seri (SAE)
    2) Numero di partecipanti con variazione rispetto al basale nei parametri clinici di laboratorio
    3) Numero di partecipanti con variazione rispetto al basale negli elettrocardiogrammi (ECG)
    4) Numero di partecipanti con variazione rispetto al basale nei segni vitali
    5) Numero di partecipanti che hanno raggiunto tappe di sviluppo motorio valutato secondo i criteri dell’Organizzazione Mondiale della Sanità (OMS)
    6) Variazione rispetto al basale nel punteggio del test per neonati sui disturbi neuromuscolari del Children’s Hospital of Philadelphia (CHOP INTEND)
    7) Variazione rispetto al basale nel punteggio della scala funzionale motoria di Hammersmith espansa (HFMSE)
    8) Variazione rispetto al basale nel punteggio del modulo revisionato relativo agli arti superiori (RULM)
    9) Tempo al decesso o alla ventilazione permanente
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to Day 778
    Fino al Giorno 778
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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 No
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    United States
    Germany
    Italy
    Spain
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    E.8.9.2In all countries concerned by the trial months8
    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.1.1.1In 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: 54
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) No
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 60
    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)
    Nusinersen is an approved treatment, and continued use after completing study requirements will be at the discretion of the participant and Investigator through prescription/commercial sources.
    Nusinersen è un trattamento approvato e l'uso continuato dopo
    il completamento dei requisiti di studio sarà a discrezione del
    partecipante e sperimentatore tramite prescrizione
    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 Decision2021-04-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-25
    P. End of Trial
    P.End of Trial StatusOngoing
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