Amyotrophic Lateral Sclerosis (ALS) MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Ferrer Internacional, S.A.
Eligibility
Sex/Gender
All
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. 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 female subject who is not of reproductive potential is defined as one who: (1) has reached natural menopause (defined as 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone [FSH] levels in the postmenopausal range as determined prior to study initiation and reported in patient’s medical records , or 12 months of spontaneous amenorrhea); (2) is 6 weeks post-surgical bilateral oophorectomy with or without hysterectomy; or; (3) has undergone bilateral tubal ligation. Spontaneous amenorrhea does not include cases for which there is an underlying disease that causes amenorrhea (e.g. anorexia nervosa) • 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 true abstinence or 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 maybe required per local requirements. Acceptable methods of birth control include 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; 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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this ag
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 |
|---|---|
| Main Objective: To assess the effect of treatment with 100 mg of FAB122 on disease progression in patients with ALS;Secondary Objective: 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 ;Primary end point(s): Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R) score after 48 weeks of treatment;Timepoint(s) of evaluation of this end point: After 48 weeks of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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 <0 and =-1, change between <-1 and =-2 etc.; 10. Staging of disease progression (King’s staging system and MiToS); 11. Overall survival: Proportion of subjects alive (survival rate) after 24, 48, 60 and 72 weeks of treatment; 12. 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, 60 and 72 weeks of treatment; 13. Absolute value and change from baseline in slow vital capacity (SVC, liters) at 24, 48, 60 and 72 weeks of treatment; 14. Absolute value and change from baseline in the overall mega score | — |
Countries
Belgium, France, Germany, Ireland, Italy, Netherlands, Poland, Portugal, Russian Federation, Spain, Sweden, United Kingdom
Contacts
Public ContactProject Manager
Julius Clinical
Outcome results
None listed