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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43841   clinical trials with a EudraCT protocol, of which   7281   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2019-000374-39
    Sponsor's Protocol Code Number:ORARIALS-02
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-06-11
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    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
    Ensayo de extensión abierto, no aleatorizado, para evaluar la seguridad y
    eficacia a largo plazo de 1200 mg/día de Arimoclomol -400 mg tres veces
    al día (t.i.d.)- en sujetos con esclerosis lateral amiotrófica (ELA) que han
    completado el ensayo 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.
    Estudio de extensión de Arimoclomol en pacientes con esclerosis lateral
    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 A/S
    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.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 Project 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.5.4Telephone number004553 706909
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic Lateral Sclerosis
    La Esclerosis Lateral Amiotrófica
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Sclerosis
    La Esclerosis Lateral Amiotrófica
    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 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.
    Evaluar la seguridad a largo plazo del arimoclomol en el tratamiento de la ELA.
    E.2.2Secondary objectives of the trial
    To evaluate the long-term efficacy of arimoclomol treatment of ALS.
    Evaluar la eficacia a largo plazo del arimoclomol en el tratamiento de la ELA.
    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. On treatment with IMP defined as last dose within 2 weeks of the last visit of the blinded ORARIALS-01.
    4. Able and willing to travel to the site for the Baseline visit and in the investigator’s opinion is expected to be able to attend the clinic for the visit at Week 4.
    1. Sujeto capaz de comprender las informaciones y dispuesto a proporcionar su consentimiento informado por escrito, así como con capacidad y disposición para cumplir con los procedimientos del estudio. En el caso de incompetencia del sujeto, el consentimiento/asentimiento informado se proporcionará de acuerdo con las regulaciones y/o procedimientos locales.
    2. Sujeto que haya completado el estudio ORARIALS-01 (es decir, que haya cumplido uno de los criterios de valoración subrogados de la supervivencia (traqueotomía o PAV) o que haya completado las 76 semanas del período de tratamiento aleatorizado).
    3. Sujeto en tratamiento con el medicamento en investigación, lo que se define como última dosis en el plazo de 2 semanas desde la última visita del estudio con diseño ciego ORARIALS-01.
    4. Sujeto con capacidad y disposición para trasladarse al centro para la visita basal y que, en opinión del investigador, sea previsiblemente capaz de acudir al centro para la visita de la Semana 4.
    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, alanine aminotransferase, lactate dehydrogenase, 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. Conocimiento o sospecha de alergia o intolerancia al medicamento en investigación (arimoclomol o sus excipientes).
    2. Exposición a algún otro tratamiento en fase de investigación, medicamento de terapia avanzada (ATMP, advanced therapy medicinal product) o uso concomitante de algún otro medicamento prohibido (véase la sección 6.8).
    3. Mujeres embarazadas o en fase de lactancia o varones o mujeres que no estén dispuestos a utilizar un método anticonceptivo de alta efectividad en el caso de que no hayan sido sometidos a esterilización quirúrgica (mujeres: ligadura de trompas bilateral, ovariectomía bilateral o histerectomía; varones: vasectomía), en el caso de las mujeres participantes hasta 4 semanas después de la última dosis y, en el caso de los varones participantes, hasta 3 meses después de la última dosis. Las mujeres premenopáusicas deberán disponer de una prueba de embarazo negativa antes de la administración del medicamento del estudio. Son métodos anticonceptivos aceptables:
    a. Métodos hormonales resultantes en inhibición de la ovulación, como anticonceptivos orales, implantables, inyectables o transdérmicos, durante un mínimo de 1 ciclo completo (basándose en el período del ciclo menstrual habitual de la sujeto) antes de la administración del medicamento en investigación.
    b. Abstinencia total de relaciones sexuales desde la última menstruación antes de la administración del medicamento en investigación. (La fiabilidad de la abstinencia sexual se evaluará en relación con la duración del estudio clínico y el estilo de vida preferido y habitual del sujeto. Los métodos de abstinencia periódica [calendario, sintotérmico, post-ovulación] no se consideran métodos anticonceptivos aceptables).
    c. Dispositivo intrauterino (IUD, intrauterine device) o sistema intrauterino (IUS, intrauterine system).
    4. Cualquiera de las siguientes afecciones médicamente importantes:
    a. Enfermedad renal o hepática clínicamente importante, O resultados clínicos de laboratorio (valores ≥ 3 veces el límite superior de normalidad (ULN, upper limit of normal) en el caso de la aspartato aminotransferasa, alanina aminotransferasa y lactato deshidrogenasa, bilirrubina ≥ 2 veces el ULN o creatinina ≥ 1,5 veces el ULN).
    b. Toda afección nueva o empeoramiento de una afección existente que, en opinión del investigador, ponga en riesgo al sujeto.
    5. Todo acontecimiento adverso grave o acontecimiento adverso moderado/severo del estudio ORARIALS-01 que estuviera aún en curso en el momento del cambio del sujeto al estudio ORARIALS-02 y que se considere probablemente relacionado con el medicamento en investigación
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence and severity of TEAEs over a treatment period of 76 weeks
    2. Mean and change from Baseline (of the present trial) to Week 76 (or end of trial) in clinical safety laboratory tests and vital signs
    3. Incidence of potentially clinically significant abnormalities in clinical safety laboratory tests and vital signs over a treatment period of 76 weeks
    4. C-SSRS over a treatment period of 76 weeks
    • Incidencia y severidad de los acontecimientos adversos surgidos durante el tratamiento (TEAE, treatment-emergent adverse event) durante un período de tratamiento de 76 semanas
    • Valor medio y variación entre el momento basal (del presente estudio) y la Semana 76 (o final del estudio) de las pruebas de laboratorio de seguridad y de las constantes vitales
    • Incidencia de las anomalías con posible significación clínica de las pruebas de laboratorio de seguridad y de las constantes vitales, durante un período de tratamiento de 76 semanas
    • Escala C-SSRS durante un período de tratamiento de 76 semanas
    E.5.1.1Timepoint(s) of evaluation of this end point
    76 weeks (or end-of-trial)
    76 semanas (o Final del estudio)
    E.5.2Secondary end point(s)
    1. Time to PAV/tracheostomy/death (for subjects entering this trial having completed 76 weeks of randomised treatment in ORARIALS-01)
    2. Change in ALSFRS-R from Baseline (of the present trial) to the end of the trial
    3. 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)
    • Tiempo hasta PAV/traqueotomía/muerte (en los sujetos participantes en este estudio que hayan completado 76 semanas de tratamiento aleatorizado en el estudio ORARIALS-01)
    • Variación de la escala ALSFRS-R entre el momento basal (del presente estudio) y el final del estudio
    • Variación de la capacidad vital lenta (SVC, slow vital capacity) entre el momento basal (del presente estudio) y el final del estudio (en los sujetos que no hayan cumplido el criterio de valoración de supervivencia en el estudio ORARIALS-01)
    E.5.2.1Timepoint(s) of evaluation of this end point
    76 weeks (or end-of-trial)
    76 semanas (o Final del estudio)
    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. 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 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.8.2.4Number 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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    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
    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 state8
    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.
    Para asegurar el tratamiento apropiado de los sujetos después de que hayan completado el ensayo, los sujetos serán tratados a criterio del investigador o remitidos a otro(s) médico(s) de acuerdo con la práctica habitual
    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 Decision2019-08-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-06
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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