Disease of nerve cells that control muscles Neurodegenerative syndrome
Conditions
Interventions
Patients will take study medication daily for the entire duration of the study,
from day 1 up to a maximum of 72 weeks. It concerns a fasting daily dose of 100
mg of FAB122 granules for oral solutio
Sponsors
Ferrer Internacional, S.A.
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
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. Slow Vital Capacity (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. * 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. 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: hormonal contraception (oral, implanted, injected or other hormonal (e.g., patch or contraceptive ring) contraception), intrauterine device in place for *3 months, barrier method in conjunction with spermicide OR use of appropriate double barrier contraception as per local regulations or guidelines; 9. Capable of providing informed consent and complying with trial procedures.
Exclusion criteria
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. Depend on mechanical ventilation (invasive or non-invasive) or require tracheostomy at Screening; 9. Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation; 10. Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, 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. Unstable psychiatric illness defined 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; 15. Current substance or alcohol dependence.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale * Revised (ALSFRS-R) score after 48 weeks of treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| Key secondary endpoints; 1. Combined assessment of function and survival (CAFS) at 48 and 72 weeks of treatment; 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 of treatment. Efficacy; 1. Change from baseline in ALSFRS-R score after 12, 24, 60, 72 weeks of treatment; 2. The slope of the decrease in ALSFRS-R score over time at 24, 48, 60 and 72 weeks of treatment; 3. Absolute ALSFRS-R score per assessment time point; 4. Change from baseline in ALSFRS-R score on Bulbar function (question 1-3 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment; 5. Change from baseline in ALSFRS-R score on Fine motor function (question 4-6 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment; 6. Change from baseline in ALSFRS-R score on Gross motor function (question 7-9 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment; 7. Change from baseline in ALSFRS-R score on Respiratory function (question 10-12 of the ALSFRS-R) after 12, 24, 60 and 72 weeks of treatment; 8. Time to a 3, 6, 9 and 12 points change from baseline in ALSFRS-R score; 9. Proportion of subjects with change from baseline in ALSFRS-R score at 24, 48, 60 and 72 weeks of treatment in categories: categories will include change *0, change between | — |
Countries
Netherlands
Outcome results
None listed