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    Summary
    EudraCT Number:2022-003050-32
    Sponsor's Protocol Code Number:FAB122-CT-2201
    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:2023-01-30
    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 number2022-003050-32
    A.3Full title of the trial
    A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
    Un estudio de extensión, multicéntrico, abierto para investigar la seguridad a largo plazo de FAB122 en pacientes con esclerosis lateral amiotrófica
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the safety of FAB122 in patients with Amyotrophic Lateral Sclerosis on the long term.
    Un estudio para investigar la seguridad de FAB122 en pacientes con esclerosis lateral amiotrófica a largo plazo.
    A.3.2Name or abbreviated title of the trial where available
    ADOREXT
    A.4.1Sponsor's protocol code numberFAB122-CT-2201
    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 SponsorFerrer Internacional, S.A.
    B.1.3.4CountrySpain
    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 supportFerrer Internacional, S.A.
    B.4.2CountrySpain
    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 code3703 CD
    B.5.3.4CountryNetherlands
    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 numberEMA/OD/184/14
    D.3 Description of the IMP
    D.3.1Product nameEdaravone
    D.3.2Product code FAB122
    D.3.4Pharmaceutical form Granules for oral solution 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 INNEdaravone
    D.3.9.1CAS number 89-25-8
    D.3.9.2Current sponsor codeFAB122
    D.3.9.4EV Substance CodeSUB06453MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 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
    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)
    Esclerosis lateral amiotrófica (ELA)
    E.1.1.1Medical condition in easily understood language
    ALS is a progressive and irreversible damage or degeneration of the nerve cells responsible for the movement of muscles.
    ELA es un daño o degeneración progresivo e irreversible de las células nerviosas responsables del movimiento de los músculos.
    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 evaluate the long-term safety of FAB122 in patients with ALS.
    Evaluar la seguridad de FAB122 a largo plazo en pacientes con ELA.
    E.2.2Secondary objectives of the trial
    1. To evaluate the effect of treatment with FAB122 on overall survival;
    2. To evaluate the effect of treatment with FAB122 on disease progression in patients with ALS;
    3. To evaluate the effect of treatment with FAB122 on cognitive functioning;
    4. To evaluate the effect of treatment with FAB122 on quality of life (QoL).
    1. Evaluar el efecto del tratamiento con FAB122 en la supervivencia general;
    2. Evaluar el efecto del tratamiento con FAB122 en la evolución de la enfermedad en pacientes con ELA;
    3. Evaluar el efecto del tratamiento con FAB122 en el funcionamiento cognitivo;
    4. Evaluar el efecto del tratamiento con FAB122 en la calidad de vida.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All patients:
    1. who completed the full study period in the main ADORE study (FAB122-CT-2001; (EudraCT number 2020-003376-40);
    2. whom the investigator has no concern and judges tolerable for the continued treatment with FAB122 from a risk and benefit point of view;
    3. a female subject should not be able to become pregnant and needs to meet at least one of the following criteria:
    - female subject who is not of reproductive potential is eligible without requiring the use of contraception. A woman is considered not having childbearing potential when becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause.
    - female who is of reproductive potential and has a negative pregnancy test at screening and at baseline and is non-lactating. A female subject who is of reproductive potential agrees to use (or have their partner use) adequate birth control methods starting from the time of consent through 30 days after the last dose of study therapy. Longer periods of birth control may be required per local requirements. Acceptable methods of birth control include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device in place for ≥3 months, intrauterine hormone-releasing system, bilateral tubal occlusion or vasectomised partner.
    4. a male patient must:
    - agree he will not donate sperm during the study and until 104 days after the last dose, AND use a condom during sexual intercourse with pregnant or non-pregnant women of childbearing potential (WOCBP) partner even if he is vasectomized.
    - in addition WOCBP partner of the male patient must use the following acceptable methods of birth control during the study and until 104 days after the last dose: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device in place for ≥3 months, intrauterine hormone-releasing system, bilateral tubal occlusion or vasectomised partner;
    5. providing informed consent.
    Todos los pacientes:
    1. que completaron todo el periodo de estudio del estudio principal ADORE (FAB122-CT-2001);
    2. cuyo estado no le preocupe al investigador y a los que considere aptos para el tratamiento continuado con FAB122 desde el punto de vista de los riesgos y beneficios.
    3. el sujeto mujer no debe quedarse embarazada y tiene que cumplir al menos uno de los siguientes criterios:
    - El sujeto mujer que no tiene capacidad reproductiva es elegible sin ser necesario el uso de anticonceptivos. Se considera que una mujer no tiene capacidad reproductiva cuando se convierte en posmenopáusica, a menos que sea permanentemente estéril. Los métodos de esterilización permanente incluyen la histerectomía, la salpingectomía bilateral y la ooforectomía bilateral. El estado posmenopáusico se define como la ausencia de menstruación durante 12 meses sin una causa médica alternativa.
    - mujeres con capacidad reproductiva que tengan una prueba de embarazo negativa en el momento del cribado y la visita inicial y que no estén lactando. Un sujeto mujer con capacidad reproductiva que se comprometa a utilizar (o hacer que su pareja utilice) métodos anticonceptivos adecuados desde el momento del consentimiento hasta 30 días después de la última dosis del tratamiento del estudio. Es posible que se requieran periodos más largos de uso de anticonceptivos según los requisitos locales. Los métodos anticonceptivos aceptables incluyen la anticoncepción hormonal combinada (que contiene estrógenos y progestágenos) asociada a la inhibición de la ovulación (oral, intravaginal, transdérmica), la anticoncepción hormonal solo de progestágenos asociada a la inhibición de la ovulación (oral, inyectable, implantable), el dispositivo intrauterino colocado durante ≥3 meses, el sistema intrauterino liberador de hormonas, la oclusión tubárica bilateral o la vasectomía de la pareja.
    4. un paciente varón debe:
    - aceptar que no donará esperma durante el estudio y hasta 104 días después de la última dosis, Y utilizar un preservativo durante las relaciones sexuales con la pareja embarazada o no embarazada en edad fértil, incluso si está vasectomizado.
    - además, la pareja en edad fértil del paciente varón debe utilizar los siguientes métodos anticonceptivos aceptables durante el estudio y hasta 104 días después de la última dosis: anticoncepción hormonal combinada (que contiene estrógenos y progestágenos) asociada a la inhibición de la ovulación (oral, intravaginal, transdérmica), anticoncepción hormonal solo de progestágenos asociada a la inhibición de la ovulación (oral, inyectable, implantable), dispositivo intrauterino colocado durante ≥3 meses, sistema intrauterino liberador de hormonas, oclusión tubárica bilateral o vasectomía de la pareja.
    5. siempre que manifiesten su consentimiento informado;
    E.4Principal exclusion criteria
    Not applicable, as study is an open-label extension of an already existing study
    No aplicable, ya que el estudio es una extensión abierto de un estudio ya existente
    E.5 End points
    E.5.1Primary end point(s)
    Nature, frequency and severity of Treatment Emergent Adverse Events.
    Naturaleza, frecuencia y gravedad de los efectos adversos emergentes del tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    As per requirement throughout whole study duration
    Según las necesidades durante toda la duración del estudio
    E.5.2Secondary end point(s)
    1. Mortality-adjusted change from baseline in ALSFRS-R total score until the end of the study;
    2. Survival time, i.e. time to death from any cause, tracheostomy or initiation of non-invasive ventilation for more than 20 hours a day for more than 10 consecutive days until the end of the study;
    3. Mean change in norm-standardized ECAS total score;
    4. Change from baseline in the total score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) until the end of the study;
    5. Change from baseline in EuroQoL – 5 Dimensions – 5 Levels (EQ-5D-5L) until the end of the study;
    6. Change from baseline in Health related QoL Visual Analogue Scale (VAS) score until the end of the study;
    7. Change from baseline in the prognostic ALS biomarker neurofilament light (NFL);
    8. Change from baseline in the ALS biomarkers creatinine and creatinine kinase;
    9. Change from baseline in the ALS biomarker Urinary extracellular domain of neurotrophin receptor p75 (Urinary P75ECD);
    10. Change from baseline of oxidative stress biomarker 8-hydroxyguanosine (8-OHdG);
    11. Cost-Utility analysis of treatment with FAB122.
    1. Cambio ajustado a la mortalidad desde el inicio en la puntuación total del ALSFRS-R hasta el final del estudio;
    2. Tiempo de supervivencia, es decir, tiempo hasta el fallecimiento por cualquier causa, traqueotomía o comenzarse a usar ventilación no invasiva durante más de 20 horas al día en un periodo de más de 10 días consecutivos, hasta el final del estudio;
    3. Cambio medio en la puntuación total en el ECAS normalizado;
    4. Cambios con respecto al inicio en la puntuación total del Formulario de evaluación de 40 preguntas de la ELA (ALSAQ-40 hasta el final del estudio;
    5. Cambios con respecto al inicio en el EuroQoL - 5 Dimensiones - 5 Niveles (EQ-5D-5L) hasta el final del estudio;
    6. Cambios con respecto al inicio en la puntuación de la escala visual analógica (EVA) en la calidad de vida en relación con la salud hasta el final del estudio;
    7. Cambios con respecto al inicio en el biomarcador pronóstico de la ELA, neurofilamentos ligeros (NFL);
    8. Cambios con respecto al inicio en los biomarcadores de la ELA creatinina y creatinina quinasa;
    9. Cambios con respecto al inicio en el biomarcador de la ELA dominio extracelular del receptor de neurotrofinas p75 en orina (P75ECD en orina).
    10. Cambios con respecto al inicio del biomarcador de estrés oxidativo: 8-hidroxiguanosina (8-OHdG);
    11. Análisis coste-utilidad del tratamiento con FAB122.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Endpoints 1, 4, 5, 6 and 11: Every 3 months
    Endpoint 2: As per requirement throughout whole study duration
    Endpoint 3: Every 6 months
    Endpoints 7, 8, 9 and 10: Every 12 months
    Criterios de valoración 1, 4, 5, 6 y 11: cada 3 meses
    Criterio de valoración 2: según las necesidades durante toda la duración del estudio
    Criterio de valoración 3: cada 6 meses
    Criterios de valoración 7, 8, 9 y 10: cada 12 meses
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    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 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 Yes
    E.8.1.7.1Other trial design description
    Estudio de extensión abierto
    Open-label extension study
    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.8.3 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA34
    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
    France
    Poland
    Sweden
    Netherlands
    Spain
    Germany
    Italy
    Belgium
    Ireland
    Portugal
    Russian Federation
    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
    Marketing Authorization of IMP or prematurely in case the objectives of the main study (ADORE, EudraCT number: 2020-003376-40) are not met.
    Autorización de comercialización del PEI o prematuramente en caso de que no se cumplan los objetivos del estudio principal (ADORE, número EudraCT: 2020-003376-40).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 147
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 78
    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 state54
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 207
    F.4.2.2In the whole clinical trial 225
    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
    Ninguno
    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 Decision2023-01-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-24
    P. End of Trial
    P.End of Trial StatusOngoing
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