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    Summary
    EudraCT Number:2019-000374-39
    Sponsor's Protocol Code Number:ORARIALS-02
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-01-27
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-000374-39
    A.3Full title of the trial
    Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial
    Studio di estensione in aperto, non randomizzato, per valutare la sicurezza e l’efficacia a lungo termine di Arimoclomol 1200 mg/giorno (400 mg tre volte al giorno, [tid.]) in soggetti affetti da sclerosi laterale amiotrofica (SLA) che hanno completato lo studio ORARIALS-01
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension study of Arimoclomol in patients with amyotrophic lateral sclerosis.
    Studio di estensione su Arimoclomol in pazienti affetti da sclerosi laterale amiotrofica.
    A.3.2Name or abbreviated title of the trial where available
    na
    na
    A.4.1Sponsor's protocol code numberORARIALS-02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03836716
    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 SponsorORPHAZYME APS
    B.1.3.4CountryDenmark
    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 supportOrphazyme A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOrphazyme A/S
    B.5.2Functional name of contact pointSenior Clinical Trial Manager
    B.5.3 Address:
    B.5.3.1Street AddressOle Maaløes Vej 3
    B.5.3.2Town/ cityCopenhagen N
    B.5.3.3Post codeDK-2200
    B.5.3.4CountryDenmark
    B.5.4Telephone number004531391062
    B.5.6E-mailhda@orphazyme.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/06/406
    D.3 Description of the IMP
    D.3.1Product nameArimoclomol
    D.3.2Product code [BRX-345]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNARIMOCLOMOL
    D.3.9.1CAS number 368860-21-3
    D.3.9.2Current sponsor codeBRX-345
    D.3.9.4EV Substance CodeSUB187159
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 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
    Amyotrophic Lateral Sclerosis
    Sclerosi Laterale Amiotrofica
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Sclerosis
    Sclerosi Laterale Amiotrofica
    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 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
    To assess the long-term safety of arimoclomol treatment of ALS.
    Valutare la sicurezza a lungo termine del trattamento a base di arimoclomol nella SLA.
    E.2.2Secondary objectives of the trial
    To evaluate the long-term efficacy of arimoclomol treatment of ALS.
    Valutare l’efficacia a lungo termine del trattamento a base di arimoclomol nella SLA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures.
    2. Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomised treatment period).
    3. Subject completed ORARIALS-01 while on treatment, where on treatment is defined as having taken the last dose of IMP within 2 weeks of the End of Trial visit. (whether at week 76 or prior).
    1. Il soggetto è in grado di comprendere ed è disposto a fornire il consenso informato scritto ed è in grado e disposto a rispettare le procedure dello studio oppure, nel caso in cui il soggetto fosse incapace, il consenso informato/assenso è fornito in conformità con le normative e/o procedure locali.
    2. Il soggetto ha completato lo studio ORARIALS-01 (cioè, ha soddisfatto uno degli endpoint surrogati di sopravvivenza di tracheotomia o PAV o ha completato il periodo di trattamento randomizzato di 76 settimane).
    3. Il soggetto ha completato ORARIALS-01 mentre era in trattamento dove essere in trattamento è definito come avere assunto l’ultima dose del farmaco sperimentale entro 2 settimane dalla visita di termine studio (alla settimana 76 o prima).
    E.4Principal exclusion criteria
    1. Known or suspected allergy or intolerance to the IMP (arimoclomol or constituents).
    2. Exposure to any other investigational treatment, advanced therapy medicinal product(ATMP) or use of any other prohibited concomitant medications.
    3. Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication. Acceptable methods of birth control are:
    a. Hormonal methods associated with inhibition of ovulation such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the subject's usual menstrual cycle period) before IMP administration.
    b. Total abstinence from sexual intercourse since the last menses before IMP administration. (The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject. Periodic abstinence methods [calendar, symptothermal, post-ovulation methods] are not acceptable methods of contraception).
    c. Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
    4. Any of the following medically significant conditions:
    a. Clinically significant renal or hepatic disease OR clinical laboratory assessment (results = 3 times the upper limit of normal [ULN] for aspartate aminotransferase, and/or alanine aminotransferase, bilirubin = 2 times the ULN, or creatinine = 1.5 times the ULN).
    b. Any new condition or worsening of existing condition which, in the opinion of the investigator would put the subject at undue risk.
    5. Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP.
    1. Allergia o intolleranza nota o sospetta al farmaco sperimentale (arimoclomol o i suoi componenti).
    2. Esposizione a qualsiasi altro trattamento sperimentale, prodotti medicinali per terapie avanzate (ATMP) o uso di altri farmaci concomitanti vietati (vedere la sezione 6.8)
    3. Donne in allattamento o in stato di gravidanza, o donne o uomini non disposti a utilizzare un metodo di controllo delle nascite altamente efficace se non chirurgicamente sterili (definito come legatura bilaterale delle tube, ooforectomia bilaterale o isterectomia per le donne; vasectomia per gli uomini) per le partecipanti di sesso femminile fino a 4 settimane dopo l’ultima dose e per i partecipanti di sesso maschile fino a 3 mesi dopo l'ultima dose. Le donne in premenopausa devono sottoporsi a un test di gravidanza con esito negativo prima di assumere il farmaco sperimentale. Metodi di controllo delle nascite accettabili sono:
    a. metodi ormonali associati ad inibizione dell’ovulazione come contraccettivi orali, impiantabili, iniettabili o transdermici per almeno 1 ciclo completo (in base al normale ciclo mestruale del soggetto) prima della somministrazione del farmaco sperimentale.
    b. Astinenza totale da rapporti sessuali a partire dalle ultime mestruazioni prima della somministrazione del farmaco sperimentale. (L’affidabilità dell’astinenza sessuale deve essere valutata in base alla durata dello studio e allo stile di vita abituale e preferito del soggetto. I metodi di astinenza periodica [calendario, metodi sintotermici, post-ovulazione] non sono metodi contraccettivi accettabili).
    c. IUD o IUS.
    4. Una qualsiasi delle seguenti condizioni clinicamente significative:
    a. Malattia renale o epatica clinicamente significativa, OPPURE valutazione clinica di laboratorio (risultati = 3 volte il limite superiore della norma [ULN] per aspartato aminotransferasi e/o alanina aminotransferasi, bilirubina = 2 volte l'ULN, o creatinina = 1,5 volte l'ULN).
    b. Qualsiasi nuova condizione o peggioramento delle condizioni esistenti che, secondo il parere dello sperimentatore,metterebbe il soggetto a rischio indebito.
    5. Qualsiasi evento avverso grave o evento avverso moderato/severo derivante dallo studio ORARIALS-01 in corso al momento della transizione a ORARIALS-02 e valutato come probabilmente associato al medicinale sperimentale.
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence and severity of TEAEs over a treatment period of 76 weeks
    • Mean and change from Baseline (of the present trial) to Week 76 (or end of trial) in clinical safety laboratory tests and vital signs
    • Incidence of potentially clinically significant abnormalities in clinical safety laboratory tests and vital signs over a treatment period of 76 weeks
    • C-SSRS over a treatment period of 76 weeks
    • Incidenza e gravità degli eventi avversi emergenti dal trattamento (TEAE) in un periodo di trattamento di 76 settimane
    • Media e variazioni dal basale (del presente studio) alla settimana 76 (o al termine dello studio) relativi ai test di laboratorio sulla sicurezza clinica e ai parametri vitali
    • Incidenza di potenziali anomalie clinicamente significative nei test di laboratorio sulla sicurezza clinica e nei parametri vitali in un periodo di trattamento di 76 settimane
    • C-SSRS in un periodo di trattamento di 76 settimane
    E.5.1.1Timepoint(s) of evaluation of this end point
    76 weeks (or end-of-trial)
    76 settimane (o al termine dello studio)
    E.5.2Secondary end point(s)
    • Time to PAV/tracheostomy/death (for subjects entering this trial having completed 76 weeks of randomised treatment in ORARIALS-01)
    • Change in ALSFRS-R from Baseline (of the present trial) to the end of the trial
    • Change in SVC from Baseline (of the present trial) to the end of the trial (for subjects who did not meet the survival endpoint in the ORARIALS-01 trial)
    • Tempo alla PAV/tracheotomia/decesso (per i soggetti che hanno partecipato a questo studio dopo aver completato 76 settimane di trattamento randomizzato in ORARIALS-01)
    • Variazione della ALSFRS-R dal basale (del presente studio) fino al termine dello studio
    • Variazione della SVC dal basale (del presente studio) fino al termine dello studio (per i soggetti che non hanno raggiunto l’endpoint di sopravvivenza nello studio ORARIALS-01)
    E.5.2.1Timepoint(s) of evaluation of this end point
    76 weeks (or end-of-trial)
    76 settimane (o al termine dello studio)
    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 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) 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 Yes
    E.8.1.7.1Other trial design description
    in aperto
    open-label
    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 EEA18
    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
    Canada
    United States
    Belgium
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Sweden
    Switzerland
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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 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: 160
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 71
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 132
    F.4.2.2In the whole clinical trial 231
    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)
    To ensure appropriate treatment of the subjects after they have completed the trial, the subjects will be treated at the investigator's discretion or referred to other physician(s) according to standard practice.
    Per garantire un trattamento appropriato dei soggetti dopo aver completato la sperimentazione, i soggetti saranno trattati a discrezione dello sperimentatore o indirizzati ad altri medici secondo la pratica standard.
    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 Decision2019-05-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-15
    P. End of Trial
    P.End of Trial StatusCompleted
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