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    Summary
    EudraCT Number:2016-003225-41
    Sponsor's Protocol Code Number:233AS102
    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:2021-06-15
    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 number2016-003225-41
    A.3Full title of the trial
    An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation
    Studio di estensione per valutare la sicurezza a lungo termine, la tollerabilità, la farmacocinetica e gli effetti sulla progressione della malattia di BIIB067 somministrato in adulti precedentemente trattati affetti da sclerosi laterale amiotrofica causata da mutazione nel gene codificante la superossido dismutasi 1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-Term Evaluation of BIIB067
    Valutazione a lungo termine di BIIB067
    A.3.2Name or abbreviated title of the trial where available
    na
    na
    A.4.1Sponsor's protocol code number233AS102
    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
    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 No
    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 namend
    D.3.2Product code [BIIB067 (ISIS666853)]
    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.3.9.2Current sponsor codeBIIB067 (ISIS 666853)
    D.3.9.4EV Substance CodeSUB179869
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6700
    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 typesecond-generation chimeric 2' methoxyethyl mixed backbone antisense oligonucleotide
    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 placeboSolution for injection
    D.8.4Route of administration of the placeboIntrathecal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic Lateral Sclerosis (ALS)
    sclerosi laterale amiotrofica (SLA)
    E.1.1.1Medical condition in easily understood language
    Lou Gehrig's disease
    Malattia di Lou Gehrig
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10077024
    E.1.2Term Familial amyotrophic lateral sclerosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    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 the study is to evaluate the long-term safety
    and tolerability of BIIB067 in participants with ALS and confirmed
    superoxide dismutase 1 (SOD-1) mutation.
    L’obiettivo primario dello studio è di valutare la sicurezza e la tollerabilità a lungo termine di BIIB067 in soggetti con SLA e mutazione SOD1 confermata.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to evaluate the pharmacokinetic (PK) and
    pharmacodynamic (PD) profiles and effects on disease progression of
    BIIB067 administered to subjects with ALS and confirmed SOD1
    mutation.
    Gli obiettivi secondari sono quelli di valutare sia i profili farmacocinetici (PK) e farmacodinamica PD che gli effetti sulla progressione della malattia di BIIB067 somministrato a soggetti con SLA e mutazione SOD1 confermata
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Must have diagnosis of SOD1-ALS, and must have completed the End of
    Study Visit for either Parts A, Band/or C of Study 233AS101 (i.e., were
    not withdrawn).
    - If taking riluzole, must be receiving a stable dose for =30 days prior to
    Day 1 and expected to remain at that dose until the final study visit.
    - For female participants of childbearing potential must agree to practice
    effective contraception during the study and for 5 months after their last
    dose of study treatment.
    - Medically able to undergo the study procedures, and to adhere to the
    visit schedule at the time of study entry, as determined by the
    Investigator.
    - Participants from Study 233AS101 Parts A and B must have a washout
    =16 weeks between the last dose of study treatment received in Study
    233AS101 and the first dose of BIIB067 received in the current Study
    233AS102.
    - If taking edaravone, participant must have initiated edaravone = 60
    days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing
    days.
    - Devono presentare una diagnosi di SLA-SOD1 e aver completato la Visita di fine studio per le parti A, B e/o C dello Studio 233AS101 (ovvero, non essere stati ritirati).
    - Se assumono riluzolo, devono averlo fatto mantenendo un dosaggio stabile per =30 giorni prima del Giorno 1 e continuare con tale dosaggio fino alla visita finale dello studio.
    - Le partecipanti in età fertile devono accettare di utilizzare un metodo contraccettivo efficace nel corso dello studio e per 5 mesi dopo la loro ultima dose di trattamento dello studio.
    - Essere in grado, dal punto di vista medico, di sottoporsi alle procedure dello studio e di aderire al programma delle visite al momento dell’ingresso nello studio, in base alle decisioni dello Sperimentatore.
    - I partecipanti provenienti dalle Parti A e B dello Studio 233AS101 devono sottoporsi a un periodo di washout =16 settimane tra l’ultima dose del trattamento dello studio ricevuta nello Studio 233AS101 e la prima dose di BIIB067 ricevuta nel presente Studio 233AS102.
    - Se assumono edaravone, i partecipanti devono avere iniziato la terapia con edaravone =60 giorni (2 cicli di trattamento) prima del Giorno 1. Edaravone non può essere somministrato nei giorni di somministrazione.
    E.4Principal exclusion criteria
    - History of allergies to a broad range of anaesthetics.
    - Presence of risk for increased or uncontrolled bleeding and/or risk of
    bleeding that is not managed optimally and could place a participant at
    an increased risk for bleeding during or after an LP procedure. These
    risks could include, but are not limited to, anatomical factors at or near
    the LP site (e.g., vascular abnormalities, neoplasms, or other
    abnormalities) and underlying disorders of the coagulation cascade,
    platelet function, or platelet count (e.g., hemophilia, Von Willebrand's
    disease, liver disease).
    - Presence of an implanted shunt for the drainage of CSF or an implanted
    CNS catheter.
    - Prior or current treatment with small interfering ribonucleic acid
    (RNA), stem cell therapy, or gene therapy.
    - Treatment with another investigational drug, biological agent
    (excluding BIIB067), or device within 1 month or 5 half-lives of study
    agent, whichever is longer.
    - Current or anticipated need, in the opinion of the Investigator, of a
    diaphragm pacing system (DPS) during the study period.
    - Female subjects who are pregnant or currently breastfeeding.
    - Current enrollment in any other interventional study.
    - Current or recent (within 1 month) use, or anticipated need, in the
    opinion of the Investigator, of copper (II) (diacetyl-bis (N4-
    methylthiosemicarbazone)) or pyrimethamine.
    NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
    - Anamnesi di allergie a una vasta gamma di anestetici.
    - Presenza di un rischio di sanguinamento maggiore o incontrollato e/o non gestito in
    modo ottimale e che potrebbe esporre il partecipante a un rischio maggiore di emorragia
    durante o dopo una procedura di PL. Questi rischi possono includere, a titolo meramente
    esemplificativo, fattori anatomici nella sede della PL o nelle sue vicinanze (es.
    anomalie vascolari, neoplasmi o altre anomalie) e patologie sottostanti riferibili alla
    cascata della coagulazione, alla funzione piastrinica o alla conta piastrinica (es.
    emofilia, malattia di Von Willebrand, patologia del fegato).
    - Presenza di shunt impiantato per il drenaggio di LCS o catetere SNC impiantato.
    - Trattamento pregresso o attuale con piccoli RNA interferenti, terapia con cellule
    staminali o terapia genetica.
    - Trattamento con un altro farmaco, agente biologico (eccetto BIIB067) o dispositivo
    sperimentale entro 1 mese o 5 emivite dell’agente dello studio, a seconda di quale sia
    più lungo.
    - Bisogno attuale o previsto, a giudizio dello Sperimentatore, di uno stimolatore
    diaframmatico (DPS) durante il periodo dello studio.
    - Pazienti di sesso femminile in gravidanza o attualmente in allattamento.
    - Arruolamento concomitante in un altro studio interventistico.
    - Uso attuale o recente (entro 1 mese) o esigenza prevista, a giudizio dello
    Sperimentatore, di rame (II) (diacetil-bis[N4-metiltiosemicarbazone]) o pirimetammina.

    NOTA: sono applicabili altri criteri di esclusione/inclusione definiti dal protocollo
    E.5 End points
    E.5.1Primary end point(s)
    Numero di partecipanti con eventi avversi ed eventi avversi gravi
    Number of participants experiencing AEs and serious adverse events
    (SAEs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to Week 248
    Fino alla Settimana 248
    E.5.2Secondary end point(s)
    - PK parameter of BIIB067 in plasma: Maximum observed concentration (Cmax)
    - PK parameter of BIIB067 in plasma: Time to reach the maximum observed concentration
    (Tmax)
    - PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time
    0 to infinity (AUCinf)
    - PK parameter of BIIB067 in plasma: Area under the concentration-time curve from time
    0 to time of the last measurable (AUClast)
    - PK parameter of BIIB067 in plasma: Apparent terminal elimination half-life (t½)
    - PK parameter of BIIB067 in CSF: Apparent terminal elimination half-life (t½)
    - Change from Baseline in ALS Functional Rating Scale – Revised (ALSFRS-R) Score
    - Change from Baseline in Slow Vital Capacity (SVC)
    - Change from Baseline in Handheld Dynamometry (HHD) Mega Score and Individual Muscle
    Strenght
    - Overall Survival
    - Ventilation Assistance-Free Survival (VAFS)
    - PD Parameter of BIIB067 in CSF: Change in Baseline in Total SOD1
    - PD Parameter of BIIB067 in CSF: Change from Baseline in Total Phosphorylated Axonal
    Neurofilament Heavy Chain (p-NHF)
    - PD Parameter of BIIB067 in Plasma: Change from Baseline in Total p- NHF
    - Parametro PK di BIIB067 nel plasma: concentrazione massima osservata (Cmax)
    - Parametro PK di BIIB067 nel plasma: tempo per raggiungere la concentrazione massima
    osservata (Tmax)
    - Parametro PK di BIIB067 nel plasma: area sotto la curva di concentrazione-tempo dal
    tempo 0 a infinito (AUCinf)
    - Parametro PK di BIIB067 nel plasma: area sotto la curva di concentrazione-tempo dal
    tempo 0 al momento dell’ultima concentrazione misurabile (AUClast)
    - Parametro PK di BIIB067 nel plasma: emivita di eliminazione terminale apparente (t½)
    - Parametro PK di BIIB067 in LCS: emivita di eliminazione terminale apparente (t½)
    - Variazione rispetto al basale del punteggio sulla Scala di valutazione funzionale
    della SLA - Rivista (ALSFRS-R)
    - Variazione rispetto al basale della capacità vitale lenta (SVC)
    - Variazione rispetto al basale del punteggio misurato mediante dispositivo per
    dinamometria manuale (HHD) e Individual Muscle Strenght
    - Sopravvivenza complessiva
    - Sopravvivenza libera da ventilazione assistita (VAFS)
    - Parametro PD di BIIB067 in LCS: variazione rispetto al basale della SOD1 totale
    - Parametro PD di BIIB067 in LCS: variazione rispetto al basale del neurofilamento
    fosforilato a catene pesanti dell’assone (p-NHF) totale
    - Parametro PD di BIIB067 nel plasma: variazione rispetto al basale del p-NHF totale
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to Week 248
    Fino alla Settimana 248
    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 Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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
    Australia
    Canada
    Japan
    United States
    European Union
    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
    The end of study is last subject, last telephone contact (approximately 24
    hours after the Final or Early Termination [Week 248]) visit. The last
    Maintenance Dose Visit for a subject will occur at Week 236, OR when
    the last subject enrolled has had their Week 92 Maintenance Dose Visit,
    whichever occurs first
    La fine dello studio è l'ultimo contatto telefonico (circa 24 ore dopo la visita
    finale o anticipata [settimana 248]) dell’ultimo soggetto. L’ultima visita alla dose
    di mantenimento per un soggetto avverrà alla settimana 236, O quando l'ultimo soggetto
    arruolato è stato visitato per la dose di mantenimento della settimana 92, a seconda
    di quale evento si verifichi per primo
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days30
    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: 146
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 37
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 38
    F.4.2.2In the whole clinical trial 182
    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
    nessuno
    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 Decision2019-12-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-10
    P. End of Trial
    P.End of Trial StatusOngoing
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