ALS muscle disease
Conditions
Interventions
TW001 is an oral solution with a concentration of 20 mg/mL in
polytethyleneglycol (PEG) 400. The solution is viscous and clear. The
administration will be performed by aspiration of TW001 with a syr
Sponsors
Treeway B.V.
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Provision of written informed consent and compliance with study procedures 2. Diagnoses of definite, probable or possible ALS according to the El Escorial criteria, supported by laboratory values and confirmed by a trained and qualified expert physician 3. Aged between 18 and 70 years of age (both inclusive) 4. Women must not be able to become pregnant (post-menopausal for at least a year, surgically sterile or practicing adequate birth control methods) for the duration of the study 5. Women of childbearing potential must have a negative serum pregnancy test at screening and be non-lactating 6. Vital capacity equal to or more than 70% predicted normal value for gender, height and age at screening 7. No clinically significant abnormality on 12-lead ECG performed at screening
Exclusion criteria
Exclusion criteria: 1. Other causes of neuromuscular weakness have not been excluded 2. Significant cognitive impairment, clinical dementia or psychiatric illness 3. Diagnosis of other neurodegenerative disease 4. Significant pulmonary disorder not attributed to ALS, requirement of treatment complicating evaluation of ALS on respiratory function 5. History of known sensitivity or intolerability of edaravone or to any related compound 6. Severe disturbance of consciousness 7. Hepatic impairment as indicated by Child-Pugh Class B or C (moderate to severe hepatic insufficiency) 8. Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation using serum creatinine. 9. Serious difficulty swallowing and/or inability to eat high-fat high-calorie meal 10. Tracheostomy or non-invasive ventilation 11. Administration of antibiotics, CYP3A4 and or PgP inhibitors 12. Smoking 13. Advanced cancer 14. Exposure to any investigational drug within 30 days of the screening visit 15. Malnourishment based on laboratory data, dehydration or on a sodium free diet 16. Presence of any of the following clinical conditions: a. Substance abuse within the past year b. Uncontrolled cardiac, pulmonary, renal, hepatic, endocrine, hematologic or active infectious disease c. AIDS or AIDS related disease, hepatitis B virus (HBV) or hepatitis C virus (HCV) d. Unstable psychiatric illness defined as psychosis, untreated major depression within 90 days of the screening visit
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. To establish the pharmacokinetic profile of a single dose TW001 2. To evaluate the effect of single dose TW001 on oxidative stress parameters in CSF (3NT) and blood (3NT, calcium, albumin, creatinine) 3. To evaluate the effect of food on the PK of TW001 and oxidative stress | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. To evaluate the safety and tolerability of TW001 2. To investigate the PK/PD relationship | — |
Countries
Netherlands
Outcome results
None listed