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. who completed the full study period in the main ADORE study (FAB122-CT-2001); 2. whom the investigator has no concern and judges tolerable for receiving treatment with FAB122 from a risk and benefit point of view; 3. a female subject should not be able to become pregnant up to 30 days after the last dose of FAB122 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 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), progestogenonly 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 period he will be using FAB122, AND use a condom during sexual intercourse with pregnant or non-pregnant women of childbearing potential (WOCBP) partner even if he is vasectomized and until 104 days after the last dose. - 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. For patients who do not take FAB122 but are only followed up by phone, only inclusion criteria #1 and #5 will apply. 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 age range 147 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 78
Exclusion criteria
Exclusion criteria: 1. Patient who has a medical condition (e.g. cardiac, pulmonary, gastrointestinal, musculoskeletal, or psychiatric illness) or personal circumstances which, in the opinion of the investigator, will make initiation or continuation of treatment with FAB122 not tolerable for them from a risk and benefit point of view. 2. Patient who discontinued study drug prematurely in the double-blind phase of the study (ADORE Study) for safety reasons. 3. Patient who has received any other investigational drug within the period between last visit of the main study and first visit of the extension study (i.e. another trial, managed access program, open label extension or early access program) 4. History of known hypersensitivity to edaravone or to any of the excipients.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety of FAB122 in patients with ALS.;Secondary Objective: 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). ;Primary end point(s): Nature, frequency and severity of Treatment Emergent Adverse Events.;Timepoint(s) of evaluation of this end point: As per requirement throughout whole study duration | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Mortality-adjusted change from baseline in ALSFRS-R total score until the end of the study; 2. Overall survival, defined as time to death from any cause or respiratory insufficiency (insufficiency defined as tracheostomy or the use of non-invasive ventilation for =20 h per day for =10 consecutive days; 3. Change from baseline in SVC until the end of the study; 4. Mean change in norm-standardized ECAS total score; 5. Change from baseline in the total score on the ALS Assessment Questionnaire-40-Item (ALSAQ-40) until the end of the study; 6. Change from baseline in EuroQoL – 5 Dimensions – 5 Levels (EQ-5D-5L) until the end of the study; 7. Change from baseline in Health related QoL Visual Analogue Scale (VAS) score until the end of the study; 8. Change from baseline in the prognostic ALS biomarker neurofilament light (NFL); 9. Change from baseline in the ALS biomarkers creatinine and creatinine kinase; 10. Change from baseline in the ALS biomarker Urinary extracellular domain of neurotrophin receptor p75 (Urinary P75ECD); 11. Change from baseline of oxidative stress biomarker 8-hydroxyguanosine (8-OHdG); 12. Cost-Utility analysis of treatment with FAB122. ;Timepoint(s) of evaluation of this end point: Endpoints 1, 5, 6, 7 and 12: Every 3 months Endpoint 2: As per requirement throughout whole study duration Endpoint 4: Every 6 months Endpoints 8, 9, 10 and 11: Every 12 months | — |
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