Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2021-000250-26
    Sponsor's Protocol Code Number:A35-004
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-23
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-000250-26
    A.3Full title of the trial
    A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS)
    Un ensayo de fase III, aleatorizado, doble ciego, controlado por placebo y multicéntrico para evaluar la seguridad y eficacia de AMX0035 frente al placebo durante un tratamiento de 48 semanas en pacientes adultos con esclerosis lateral amiotrófica (ELA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the safety and efficacy of AMX0035 in patients with Amyotrophic Lateral Sclerosis
    Un estudio para investigar la seguridad y eficacia de AMX0035 en pacientes con Esclerosis Lateral Amiotrófica
    A.3.2Name or abbreviated title of the trial where available
    Phoenix
    Phoenix
    A.4.1Sponsor's protocol code numberA35-004
    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 SponsorAmylyx Pharmaceuticals Inc.
    B.1.3.4CountryUnited States
    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 supportAmylyx Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJulius Clinical
    B.5.2Functional name of contact pointProject Manager
    B.5.3 Address:
    B.5.3.1Street AddressBroederplein 41-43
    B.5.3.2Town/ cityZeist
    B.5.3.3Post code3703CD
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31306569900
    B.5.5Fax number+31306569990
    B.5.6E-mailregulatory@juliusclinical.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/3/20/2284
    D.3 Description of the IMP
    D.3.2Product code AMX0035
    D.3.4Pharmaceutical form Oral powder in sachet
    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 INNfenilbutirato
    D.3.9.1CAS number 1716-12-7
    D.3.9.4EV Substance CodeSUB127264
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNUrsodoxicoltaurina
    D.3.9.1CAS number 14605-22-2
    D.3.9.3Other descriptive nametaurursodiol
    D.3.9.4EV Substance CodeSUB218624
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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) 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 No
    D.IMP: 2
    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/3/20/2284
    D.3 Description of the IMP
    D.3.2Product code AMX0035
    D.3.4Pharmaceutical form Oral powder in sachet
    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 INNFenilbutirato
    D.3.9.1CAS number 1716-12-7
    D.3.9.4EV Substance CodeSUB127264
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNUrsodoxicoltaurina
    D.3.9.1CAS number 14605-22-2
    D.3.9.3Other descriptive nametaurursodiol
    D.3.9.4EV Substance CodeSUB218624
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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) 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 No
    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 placeboOral powder in sachet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    ALS (amyotrophic lateral sclerosis)
    ELA (esclerosis lateral amiotrófica)
    E.1.1.1Medical condition in easily understood language
    ALS
    ELA
    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
    1. To assess the safety and tolerability of AMX0035 treatment over 48 weeks compared to placebo in adult patients with ALS.
    2. To assess the impact of AMX0035 treatment compared to placebo on disease progression over 48 weeks based on change from baseline of ALSFRS-R and survival.
    1. Evaluar la seguridad y tolerabilidad del tratamiento con AMX0035 durante 48 semanas en comparación con el placebo en pacientes adultos con ELA.
    2. Evaluar el impacto del tratamiento con AMX0035, en comparación con el placebo, en el progreso de la enfermedad durante 48 semanas, basándose en el cambio desde el inicio de la ALSFRS R y la supervivencia.
    E.2.2Secondary objectives of the trial
    1. To assess the impact of AMX0035 compared to placebo on Slow Vital Capacity (SVC) over 48 weeks.
    2. To assess patient quality of life (QOL; using the 40-item ALS assessment questionnaire [ALSAQ-40] patient-reported outcome [PRO]) during treatment with AMX0035 compared to placebo over 48 weeks.
    3. To assess AMX0035 compared to placebo on the time to any decline in King’s and MiToS stages as derived from ALSFRS-R data over 48 weeks.
    4. To assess the impact of AMX0035 compared to placebo on ventilation free survival (defined as death, tracheostomy for respiratory distress or permanent non-invasive ventilation [>22 hours per day for 7 consecutive days]) over 48 weeks.
    5. To assess AMX0035 compared to placebo on patient health status (using the EQ-5D descriptive system and the EQ visual analogue scale [EQ VAS] PRO during treatment with AMX0035 over 48 weeks.
    Finally, a secondary objective of the trial is to assess the long-term survival of patients treated with AMX0035 or placebo.
    1.Evaluar el impacto del fármaco AMX0035 en comparación con el placebo en la CVL en pacientes adultos con ELA durante 48 sem.
    2.Evaluar la CV, mediante el resultado comunicado por el paciente(PRO)del cuestionario de evaluación de 40 puntos de la ELA[ALSAQ-40]durante el tratamiento vs placebo,en pacientes adultos con ELA durante 48 sem.
    3.Evaluar AMX0035 vs placebo en el tiempo para cualquier disminución en los sistemas de estadificación de King y MITOS derivados de los datos de la ALSFRS-R durante 48 sem.
    4.Evaluar impacto de AMX0035 vs placebo en la supervivencia sin ventilación en pacientes adultos con ELA durante 48 sem.
    5.Evaluar AMX0035 vs placebo en el estado de salud del paciente,mediante el uso del sistema descriptivo EQ-5D y la escala visual analógica EQ(EVA EQ)PRO durante el tratamiento de 48 sem.
    Evaluar la supervivencia general a largo plazo de los pacientes adultos con ELA tratados con MX0035 o placebo
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    A sub-study is included in the main protocol. Matched evaluation will be performed of QTc interval prolongation (evaluated by digital ECG with central read) and blood sample for evaluation of systemic exposure (measurement in plasma using a validated assay for PB and its major metabolite PAA, and taurursodiol and its major metabolites ursodiol and glycine conjugatedursodiol).
    E.3Principal inclusion criteria
    1. Male or female, at least 18 years of age.
    2. Diagnosis of ALS (definite or clinically probable), made by a physician who is experienced with management of ALS, as defined by the World Federation of Neurology revised El Escorial criteria.
    3. Time since onset of first symptom of ALS should be <24 months.
    4. If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the screening visit, started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone.
    5. Capable of providing informed consent.
    6. Capable and willing to follow trial procedures including visits to the trial clinic and visit requirements.
    7. Women of childbearing potential (WOCBP; e.g., not post-menopausal for at least one year or surgically sterile*) must agree to use adequate birth control** for the duration of the trial and 3 months after the last dose of study drug.
    8. Women must not be pregnant or planning to become pregnant for the duration of the trial and 3 months after last dose of study drug.
    9. Men must agree to practice contraception for the duration of the trial and for at least 3 months after last dose of study drug;
    10. Men must not plan to father a child or to provide sperm for donation for the duration of the trial and 3 months after the last dose of study drug.
    * 12 months of spontaneous amenorrhea or 6 months of spontaneous amenorrhea with serum follicle stimulating hormone (FSH) levels > 40 mIU/ml or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy.
    ** Acceptable contraception for use in this trial are:
    - Hormonal methods, such as birth control pills, patches, injections, vaginal ring, or implants;
    - Barrier methods (such as a condom or diaphragm) used with a spermicide (a foam, cream, or gel that kills sperm);
    - Intrauterine device (IUD);
    - Abstinence (no heterosexual sex);
    - Unique partner who is surgically sterile (men) or not of childbearing potential (female).
    1. Hombre o mujer, mayor de 18 años.
    2. Haber sido diagnosticado con ELA (definitivo o clínicamente probable) por un médico con experiencia en el tratamiento de la ELA, según lo definido por los Criterios revisados de El Escorial de la Federación Mundial de Neurología.
    3. El tiempo transcurrido desde la aparición del primer síntoma de ELA debe ser <24 meses antes de la aleatorización.
    4. Si el participante va a ser tratado con riluzol y/o edaravona durante el transcurso del ensayo, para el momento de la visita de selección el tratamiento con riluzol y/o edaravona debe haber sido previamente mantenido en un régimen estable durante al menos 14 días para el riluzol y/o un ciclo completo de tratamiento para la edaravona.
    5. Ser capaz de dar su consentimiento informado.
    6. Ser capaz y estar dispuesto a seguir los procedimientos del ensayo, incluidas las visitas a la clínica del ensayo y los requisitos de las visitas.
    7. Las mujeres en edad fértil (WOCBP), por ejemplo, que no hayan sido posmenopáusicas durante al menos un año o que no sean quirúrgicamente estériles, deben aceptar utilizar un método anticonceptivo adecuado** mientras dure el ensayo y 3 meses después de la última dosis del fármaco del estudio.
    8. Las mujeres no deben estar embarazadas ni tener la intención de quedarse embarazadas mientras dure el ensayo ni 3 meses después de la última dosis del fármaco del estudio.
    9. Los hombres deben aceptar utilizar métodos anticonceptivos mientras dure el ensayo y al menos los 3 meses posteriores a la última dosis del fármaco del estudio.
    10. Los hombres no deben planear ser padres ni donar esperma mientras dure el ensayo ni 3 meses después de la última dosis del fármaco del estudio.
    *12 meses de amenorrea espontánea o 6 meses de amenorrea espontánea con niveles séricos de FSH >40 mI U/ml o 6 semanas después de una ooforectomía bilateral con o sin histerectomía.
    **Los métodos anticonceptivos aceptados en este ensayo son:
    • Métodos hormonales, como los parches, píldoras, inyecciones, anillo vaginal o implantes anticonceptivos.
    • Métodos de barrera, como el preservativo o diafragma, utilizados con espermicida, una espuma, crema o gel que elimina los espermatozoides.
    • El dispositivo intrauterino (DIU).
    • La abstinencia (no tener relaciones sexuales heterosexuales).
    • Pareja única que es quirúrgicamente estéril (hombres) o que no está en edad fértil (mujeres).
    E.4Principal exclusion criteria
    1. Presence of tracheostomy or PAV.
    2. Slow Vital Capacity (SVC) less than 55%.
    3. History of known allergy to PB or bile salts.
    4. Abnormal liver function defined as bilirubin levels and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 5 times the upper limit of the normal (obtained within 12 weeks from first dose).
    5. Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2 (obtained within 12 weeks from first dose).
    6. Pregnant women (confirmed by a pregnancy test within 7 days of first dose) or women currently breastfeeding.
    7. Current severe biliary disease which may result in the Investigator medical judgment in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gangrene of the gallbladder, abscess of the gallbladder.
    8. History of Class III/IV heart failure (per New York Heart Association – NYHA).
    9. Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Investigator clinical judgment.
    10. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment.
    11. Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, clinically significant
    laboratory test or ECG abnormality) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment;
    12. Previous treatment for ALS with cellular therapies or gene therapies;
    13. Currently enrolled on another trial involving use of an investigational therapy;
    14. Previous treatment with PB or taurursodiol within 30 days from Screening
    15. Implantation of Diaphragm Pacing System (DPS);
    16. Current or previous treatment in the last 30 days from first dose or planned exposure to any prohibited medications listed in Section 6.8 of the protocol.
    1. Presencia de traqueotomía o PAV.
    2. Capacidad Vital Lenta (CVL) inferior al 55 %.
    3. Antecedentes de alergia conocida al fenilbutirato de sodio o a las sales biliares.
    4. Función hepática anormal definida como niveles de bilirrubina y/o aspartato aminotransferasa (AST) y/o alanina aminotransferasa (ALT) >5 veces el límite máximo de lo normal (obtenidos en las 12 semanas siguientes a la primera dosis).
    5. Insuficiencia renal según lo definido por la TFGe: <60 mL/min/1,73 m2 (obtenida en las 12 semanas siguientes a la primera dosis).
    6. Mujeres embarazadas (confirmadas con una prueba de embarazo en los 7 días siguientes a la primera dosis) o mujeres actualmente en periodo de lactancia.
    7. Enfermedad biliar actual grave que puede dar lugar, según el criterio de los investigadores, a una obstrucción biliar, incluidos, por ejemplo, la colecistitis crónica, la cirrosis biliar primaria, la colangitis esclerosante, el cáncer de vesícula biliar, la gangrena de la vesícula biliar y el absceso de la vesícula biliar.
    8. Antecedentes de insuficiencia cardíaca de clase III/IV, según la New York Heart Association (NYHA).
    9. Participante con una restricción grande del consumo de sal para el que la ingesta añadida de sal por el tratamiento supusiera un riesgo, a juicio clínico de los investigadores.
    10. Presencia de enfermedades psiquiátricas inestables, deterioro cognitivo, demencia o abuso de sustancias que perjudiquen la capacidad del participante para dar su consentimiento informado, según el criterio de los investigadores.
    11. Enfermedad inestable clínicamente significativa (que no sea la ELA), por ejemplo, inestabilidad cardiovascular, infección sistémica, disfunción tiroidea no tratada, anomalía grave en las pruebas de laboratorio o cambios en el electrocardiograma clínicamente significativos (ECG); que supondría un riesgo para el participante si él o ella formara parte del ensayo, según el criterio de los investigadores.
    12. Tratamiento previo de la ELA con terapias celulares o génicas.
    13. Actualmente inscrito en otro ensayo que implique el uso de una terapia en investigación.
    14. Tratamiento previo con fenilbutirato de sodio o taurursodiol en los 30 días siguientes a la selección.
    15. Implantación del Sistema de Estimulación del Diafragma (DPS).
    16. Actualmente en tratamiento o previamente tratado en los últimos 30 días o exposición prevista a cualquier medicamento prohibido que figure en la sección del protocolo.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Safety Endpoints:
    o Incidence and severity of AE and SAEs
    o Incidence of abnormalities in clinical laboratory assessments
    o Withdrawal from the trial
    Primary Efficacy Endpoint
    o Joint assessment of ALSFRS-R total score progression over 48 weeks adjusted for mortality
    Variables de valoración primarias de seguridad:
    o Incidencia y gravedad de los AA y AAG
    o Incidencia de las pruebas de laboratorio anormales
    o Retirada del ensayo
    Variable primaria de eficacia
    o Evaluación conjunta de la progresión de la puntuación total de la ALSFRS-R durante 48 semanas ajustada por la mortalidad
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs and SAEs will be monitored continuously.
    Clinical laboratory safety assessments will be performed at Screening and baseline, and 24 and 48 weeks after start of treatment.
    ALSFRS-R will be assessed at baseline and every 4 weeks after start of treatment until 48 weeks after start of treatment.
    Los AA y los AAG se monitorizarán de forma continua.
    Las evaluaciones de seguridad del laboratorio clínico se llevarán a cabo en el momento de la selección y en basal, y 24 y 48 semanas después del iniciar el tratamiento.
    La ALSFRS-R se evaluará en basal y cada 4 semanas después del comienzo del tratamiento hasta 48 semanas después del inicio del tratamiento.
    E.5.2Secondary end point(s)
    o Change from baseline to Week 48 of the SVC as percentage of normalized values for gender, body weight, age, and ethnicity
    o Change from baseline to Week 48 of the ALSAQ-40 total score
    o Time from baseline to transition through a King’s stage or through a MiToS stages (derived from ALSFRS-R data)
    o Time from baseline remaining ventilation free i.e., death, tracheostomy for respiratory distress, permanent non-invasive ventilation (PAV)
    o Change from baseline of the EQ-5D score and visual analog scale (VAS) at Week 48
    o Overall survival with all-cause mortality, assessed by independent confirmation of vitals status of all patients randomized into the trial extending after completion of the planned trial follow-up of 48 weeks
    o Cambio desde basal hasta la semana 48 del CVL como porcentaje de los valores normalizados para el género, el peso corporal, la edad y etnia
    o Cambio desde basal hasta la semana 48 de la puntuación total del ALSAQ-40
    o Tiempo desde basal hasta la transición a través del sistema de estadificación de King o a través de sistema de estadificación de MiToS (derivado de los datos de ALSFRS-R)
    o Tiempo desde basal en el que no hay ventilación, es decir, muerte, traqueotomía por dificultad respiratoria, Ventilación Asistida Proporcional (PAV)
    o Cambio desde basal de la puntuación EQ-5D y la escala visual analógica (EVA) en la semana 48
    o Supervivencia global con mortalidad por todas las causas, evaluada mediante la confirmación independiente del estado de las constantes vitales de todos los pacientes aleatorizados en el ensayo, que se prolonga tras la finalización del seguimiento previsto del ensayo de 48 semanas
    E.5.2.1Timepoint(s) of evaluation of this end point
    SVC will be assessed at Screening and baseline, and every 4 weeks after start of treatment until 48 weeks after start of treatment.
    ALSAQ-40, King's stage, MiToS stage, EQ-5D-score and VAS scale will be assessed at baseline and every 12 weeks after start of treatment until 48 weeks after start of treatment.
    La CVL se evaluará en la selección y basal, y cada 4 semanas después del inicio del tratamiento hasta 48 semanas después del inicio del tratamiento.
    El ALSAQ-40, los sistemas de estadificación de King, los sistemas de estadificación de MiToS, la puntuación EQ-5D y la escala EVA se evaluarán al inicio y cada 12 semanas después del inicio del tratamiento hasta 48 semanas después del inicio del tratamiento.
    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 No
    E.6.11Pharmacogenomic Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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 States
    Belgium
    France
    Germany
    Ireland
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    Sweden
    United Kingdom
    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
    The end of the trial will correspond to the last participant last EOT visit/phone contact per the trial schedule.
    El final del ensayo corresponderá a la última visita/contacto telefónico del participante según el programa del ensayo.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    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: 420
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 180
    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 state55
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 360
    F.4.2.2In the whole clinical trial 600
    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)
    Post trial, AMX0035 will be made available to participants completing the 48-week study period in accordance with each competent authority’s regulatory guidance.
    Después del ensayo, AMX0035 se pondrá a disposición de los participantes que completen el período de estudio de 48 semanas, de acuerdo con las guías regulatorias de cada autoridad competente.
    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 Decision2021-10-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-22
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Mon May 05 03:05:13 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA