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    Summary
    EudraCT Number:2022-003050-32
    Sponsor's Protocol Code Number:FAB122-CT-2201
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-11-17
    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 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
    Uno studio di estensione in aperto, multicentrico, per valutare la sicurezza a lungo termine di FAB122 in pazienti con Sclerosi Laterale Amiotrofica
    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.
    Uno studio per valutare a lungo termine la sicurezza di FAB122 nei pazienti con Sclerosi Laterale Amiotrofica.
    A.3.2Name or abbreviated title of the trial where available
    ADOREXT
    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
    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.3.10 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 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 (ALS)
    Sclerosi Laterale Amiotrofica (SLA)
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Sclerosis (ALS)
    Sclerosi Laterale Amiotrofica (SLA)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    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.
    Valutare la sicurezza a lungo termine di FAB122 in pazienti affetti da SLA.
    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. Valutare gli effetti del trattamento con FAB122 sulla sopravvivenza globale;
    2. Valutare gli effetti del trattamento con FAB122 sulla progressione della malattia nei pazienti affetti da SLA;
    3. Valutare gli effetti del trattamento con FAB122 sulle funzioni cognitive;
    4. Valutare gli effetti del trattamento con FAB122 sulla qualità di vita (QoL).
    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.
    1. aver completato l’intero periodo di studio dello nello studio principale ADORE (FAB122-CT-2001);
    2. lo sperimentatore non nutre preoccupazioni e considera ritiene tollerabile il trattamento continuato con FAB122 dal punto di vista dei rischi e benefici;
    3. i soggetti di sesso femminile non devono poter iniziare una gravidanza e devono soddisfare almeno uno dei seguenti criteri:
    - i soggetti di sesso femminile privi di potenziale riproduttivo sono idonei senza dover ricorrere all’utilizzo di contraccettivi. Una donna viene considerata non potenzialmente fertile se è in post-menopausa, a meno che non sia permanentemente sterile. I metodi di sterilizzazione permanente includono isterectomia, salpingectomia bilaterale e ovariectomia bilaterale. Uno stato post-menopausale è definito come assenza di mestruazioni da 12 mesi senza che ci siano altre cause mediche.
    - soggetti di sesso femminile con potenziale riproduttivo, con test di gravidanza negativo allo screening e alla visita basale, non in fase di allattamento. I soggetti di sesso femminile con potenziale riproduttivo devono accettare di utilizzare (o di far utilizzare al proprio partner) metodi contraccettivi adeguati a partire dal momento del consenso fino ai 30 giorni successivi all’ultima dose della terapia in studio. A seconda dei requisiti locali, potrebbe essere necessario impiegare un metodo contraccettivo per periodi più lunghi. I metodi contraccettivi accettabili comprendono i contraccettivi ormonali combinati (contenenti estrogeno e progestinico) associati a inibitori dell’ovulazione (per via orale, intravaginale o transdermica), i contraccettivi ormonali a base di solo progestinico associati a inibitori dell’ovulazione (orali, iniettabili o impiantabili), un dispositivo intrauterino impiantato da = 3 mesi, un sistema intrauterino a rilascio ormonale, l’occlusione tubarica bilaterale o partner sottoposto a vasectomia.
    4. i pazienti di sesso maschile devono:
    - accettare di non donare lo sperma durante lo studio e fino a 104 giorni dopo l’ultima dose E utilizzare il preservativo durante i rapporti sessuali con donne in stato di gravidanza oppure non in stato di gravidanza e potenzialmente fertili, anche se il paziente è stato sottoposto a vasectomia.
    - inoltre, la relativa partner potenzialmente fertile deve utilizzare i seguenti metodi contraccettivi accettabili durante lo studio e fino a 104 giorni dopo l’ultima dose: contraccettivi ormonali combinati (contenenti estrogeno e progestinico) associati a inibitori dell’ovulazione (per via orale, intravaginale o transdermica), contraccettivi ormonali a base di solo progestinico associati a inibitori dell’ovulazione (orali, iniettabili o impiantabili), dispositivo intrauterino impiantato da = 3 mesi, sistema intrauterino a rilascio ormonale, occlusione tubarica bilaterale o partner sottoposto a vasectomia.
    5. fornire il consenso informato.
    E.4Principal exclusion criteria
    Not applicable, as study is an open-label extension of an already existing study.
    Non applicabile, in quanto lo studio è un'estensione in aperto di uno studio già esistente.
    E.5 End points
    E.5.1Primary end point(s)
    Nature, frequency and severity of Treatment Emergent Adverse Events.
    Natura, frequenza e gravità degli eventi avversi emergenti dal trattamento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout whole study duration.
    Per tutta la durata dello studio.
    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. Variazione aggiustata per la mortalità rispetto al basale nel punteggio totale ALSFRS-R fino al termine dello studio;
    2. Tempo di sopravvivenza, ossia il periodo di tempo fino al decesso dovuto a qualsiasi causa, tracheostomia o inizio della ventilazione non invasiva per oltre 20 ore al giorno per più di 10 giorni consecutivi fino al termine dello studio;
    3. Variazione media nel punteggio totale ECAS standardizzato;
    4. Variazione rispetto al basale nel punteggio totale per il questionario 40-item ALS Assessment Questionnaire (ALSAQ-40) fino al termine dello studio;
    5. Variazione rispetto al basale nel questionario EuroQoL – 5 Dimensions – 5 Levels (EQ-5D-5L) fino al termine dello studio;
    6. Variazione rispetto al basale nel punteggio della scala analogica visiva (VAS) per la qualità della vita correlata alla salute fino al termine dello studio;
    7. Variazione rispetto al basale del neurofilamento a catena leggera (NFL) quale biomarcatore prognostico per la SLA;
    8. Variazione rispetto al basale dei biomarcatori per la SLA creatinina e creatinina chinasi;
    9. Variazione rispetto al basale del dominio extracellulare del recettore delle neurotrofine p75 nelle urine (P75ECD urinario) quale biomarcatore per la SLA;
    10. Variazione rispetto al basale del biomarcatore di stress ossidativo 8-idrossiguanina (8-OHdG);
    11. Analisi costo-utilità del trattamento 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: Throughout whole study duration
    Endpoint 3: Every 6 months
    Endpoints 7, 8, 9 and 10: Every 12 months
    Endpoint 1, 4, 5, 6 e 11: ogni 3 mesi
    Endpoint 2: durante l'intera durata dello studio
    Endpoint 3: ogni 6 mesi
    Endpoint 7, 8, 9 e 10: ogni 12 mesi
    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 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
    Studio di estensione in aperto
    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.8.2.4Number 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 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 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
    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
    Until the marketing authorization of IMP or prematurely in case the objectives of the main study (ADORE, EudraCT number: 2020-003376-40) are not met.
    Fino alla autorizzazione all’immissione in commercio dell'IMP o anticipatamente in caso di mancato raggiungimento degli obiettivi dello studio principale (ADORE, EudraCT number: 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: 188
    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 state37
    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
    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 Decision2023-02-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-02-21
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2024-01-08
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