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    EudraCT Number:2020-003376-40
    Sponsor's Protocol Code Number:FAB122-CT-2001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-06-08
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2020-003376-40
    A.3Full title of the trial
    A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
    A.4.1Sponsor's protocol code numberFAB122-CT-2001
    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.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.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
    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. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboGranules for oral solution 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
    Amyotrophic Lateral Sclerosis
    E.1.1.1Medical condition in easily understood language
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the effect of treatment with 100 mg of FAB122 on disease progression in patients with ALS
    E.2.2Secondary objectives of the trial
    1. To evaluate the effect of treatment with FAB122 on survival
    2. To evaluate the safety and tolerability of FAB122
    3. To evaluate the effect of treatment with FAB122 on quality of life (QoL)
    4. To evaluate the effect of treatment with FAB122 on cognitive functioning
    5. To evaluate the pharmacokinetics (PK) of FAB122
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    There will be an optional PK sub-study, included in the main protocol. The objective is to evaluate the pharmacokinetics (PK) of FAB122.
    E.3Principal inclusion criteria
    1. Age 18 – 80 years (both inclusive), male or female;
    2. Diagnosis of definite, probable, probable laboratory supported or possible ALS as based on the El Escorial and the revised Airlie House diagnostic criteria for ALS;
    3. Onset of first symptoms* no longer than 24 months prior to randomization;
    *Date of onset is the date the patient reported one or more of the following symptoms: Muscle weakness in limbs; speech/swallowing difficulties; respiratory symptoms: dyspnea was noticed
    4. SVC equal to or more than 70% of the predicted normal value for gender, height and age at screening visit;
    5. Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit;
    6. Patients on riluzole should be on stable doses ≥30 days prior to the baseline visit and this dose should be maintained during the entire trial.
    7. 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. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
    • 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) or practicing 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. A vasectomised partner is a highly effective contraception method provided that the partner is the sole male sexual partner of the WOCBP and the absence of sperm has been confirmed. If not, an additional highly effective method of contraception should be used.
    8. 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.;
    9. Capable of providing informed consent and complying with trial procedures.
    E.4Principal exclusion criteria
    1. Diagnosis of Primary Lateral Sclerosis;
    2. Diagnosis of Frontotemporal Dementia;
    3. Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease, Alzheimer disease);
    4. Diagnosis of polyneuropathy;
    5. Other causes of neuromuscular weakness;
    6. Have a significant pulmonary disorder not attributed to ALS and/or require treatment interfering with the evaluation of ALS on respiratory function;
    7. Use of intravenous (IV) edaravone within 6 months of the screening visit;
    8. Use of mechanical ventilation (invasive or non-invasive) at Screening;
    9. Renal impairment as indicated by a creatinine clearance of less than 50 mL/min;
    10. Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, ALT/AST levels ≥ 3xULN, bilirubin levels ≥2xULN or subject has a positive screening test for HIV, hepatitis B or C;
    11. Presence of any of the following clinical conditions:
    a. Unstable cardiac, pulmonary, endocrine, hematologic or active infectious disease
    b. Severe active psychiatric illness e.g. as psychosis, untreated major depression within 90 days of the screening visit
    c. Significant cognitive impairment, clinical dementia or psychiatric illness
    d. Cancer that is currently under active treatment or is likely to require treatment during the trial that may alter the subject´s function and interfere with assessment of ALS disease progression.
    12. Any comorbidity that may interfere with the functions as scored with the ALSFRS-R;
    13. History of known sensitivity or intolerability to edaravone, to any related compound, or to any of the excipients;
    14. Exposure to any investigational drug within 30 days of the screening visit; or 5 half-lives, whichever is longer;
    15. Current substance or alcohol dependence.
    16. For patients undergoing optional CSF sampling: any condition that according to the investigator criteria is contraindicated for the procedure (e.g. space-occupying lesion with mass effect, increase of intracranial pressure due to increased CSF pressure; posterior fossa mass; Arnold-Chiari malformation; anticoagulant medication; coagulopathy; uncorrected bleeding diathesis; congenital spine abnormality; and skin infection at puncture site
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R) score after 48 weeks
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 48 weeks
    E.5.2Secondary end point(s)
    Key secondary endpoints
    1. Combined assessment of function and survival (CAFS) at 48 and 72 weeks;
    2. Survival time, i.e. time to death, tracheostomy or initiation of non-invasive ventilation for more than 20 hours a day for more than 10 consecutive days, over 72 weeks

    1. Change from baseline in ALSFRS-R score after 24 and 72 weeks;
    2. The slope of the decrease in ALSFRS-R score over time at 24, 48 and 72 weeks;
    3. Change from baseline in ALSFRS-R score on Bulbar function (question 1-3 of the ALSFRS-R) after 24, 48 and 72 weeks;
    4. Change from baseline in ALSFRS-R score on Fine motor function (question 4-6 of the ALSFRS-R) after 24, 48 and 72 weeks;
    5. Change from baseline in ALSFRS-R score on Gross motor function (question 7-9 of the ALSFRS-R) after 24, 48, and 72 weeks;
    6. Change from baseline in ALSFRS-R score on Respiratory function (question 10-12 of the ALSFRS-R) after 24, 48, and 72 weeks;
    7. Time to a 3, 6, 9 and 12 points change or death from baseline in ALSFRS-R score, over 72 weeks;
    8. Proportion of subjects with change from baseline in ALSFRS-R score at 24, 48, and 72 weeks in categories: categories will include change ≥0, change between <0 and ≥-1, change between <-1 and ≥-2 etc.;
    9. Time to change in clinical staging or death (King’s staging system and MiToS) over 72 weeks;
    10. Overall survival: Proportion of subjects alive (survival rate) after 24, 48, and 72 weeks;
    11. Proportion of subjects alive and no tracheostomy, or no initiation of non-invasive ventilation for more than 20 hours a day for more than 10 consecutive days after 24, 48, and 72 weeks;
    12. Change from baseline in slow vital capacity (SVC, liters) at 24, 48, and 72 weeks;
    13. Change from baseline in the overall mega score for the hand-held dynamometer (HHD) at 24, 48, and 72 weeks.

    1. Change from baseline in the total score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) Form at 24, 48 and 72 weeks;
    2. Change from baseline in EuroQoL – 5 Dimensions-5 Levels (EQ-5D-5L) questionnaire score and health related QoL at 24, 48, 60 and 72 weeks.
    3. Change from baseline in visual Analogue Scale (VAS) score at 24, 48, and 72 weeks.

    1. Proportion of subjects with a change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS (Edinburgh Cognitive and behavioural ALS Screen) total score;
    2. Change from baseline for ALS Specifi, ALS Non-Sepcific, and ECAS total score at 24, 48 adn 72 weeks;
    3. Time to a mean change of ≥8, ≥4, and ≥9 for ALS Specific, ALS Non-Specific, and ECAS total score.

    (Population) PK parameters of FAB122 and riluzole
    E.5.2.1Timepoint(s) of evaluation of this end point
    As indicated in the end points, after 12, 24, 48, 60, and/or 72 weeks
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    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. 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    Russian Federation
    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
    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 months2
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days15
    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: 250
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 50
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 250
    F.4.2.2In the whole clinical trial 300
    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)
    The Sponsor commits to offering all patients continuation of the treatment with the IMP, FAB122, after the end of the study until FAB122 becomes available, unless the Sponsor is forced to terminate the clinical development program of FAB122 for any reason, e.g. if analysis of the data fails to confirm significant treatment effects on one or more of the endpoints, or in case of any serious safety effects of the treatment with FAB122.
    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 Decision2021-08-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-10-26
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