amyotrophic lateral sclerosis amyotrophic lateral sclerosis ALS
Conditions
Sponsors
Tansy Aaron
Eligibility
Inclusion criteria
Inclusion criteria: 1. Males or females aged between 18 and 80 years with a body mass index between 18 and 35 kg/m2 at the time of the initial Screening Visit 2. Onset of the first symptom leading to the diagnosis of ALS is within 24 months at the time of the initial Screening Visit 3. Revised El Escorial criteria of either: (i) Clinically definite ALS (ii) Clinically probable ALS 4. A total ALSFRS-R score of at least 34 at the start of the Screening Period
Exclusion criteria
Exclusion criteria: 1.History of allergies or adverse reactions to any of the excipients in the study drug formulation 2. Liver failure (except in Gilbert syndrome, defined as AST and/or ALT 3 times the upper limit of normal [ULN] or liver function test [LFT] with bilirubin 1.5 times the ULN)) 3. Moderate to severe renal failure (defined as estimated glomerular filtration rate [eGFR] less than 60 mL/min) 4. Currently participating in a clinical trial of another investigational drug, or have participated 30 days prior to the start of the screening period or 5 half-lives from the most recent dose of the investigational drug, whichever is longer. 5. History of alcohol or drug abuse within 6 months prior to the start of the screening period 6. Patient has received combination therapy with edaravone and sodium phenylbutyrate/taurursodiol within 30 days prior to the start of the screening period. 7. History of breast cancer (regardless of remission status) or history of breast cancer in a first-degree relative
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is the change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) in the Intent-To-Treat 1 (ITT1) Analysis Set after 24 weeks of treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Safety and tolerability of PTC857 2. Subject's respiratory function 3. Subject's motor/limb function and medullary function 4. Neuropsychological functioning of subjects 5. Subject survival period 6. Subject's quality of life 7. Pharmacokinetics of PTC857 | — |
Countries
Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Ireland, Italy, Japan, Mexico, Netherlands, Norway, Poland, Spain, Sweden, US
Contacts
Public ContactMin Kim
Worldwide Clinical Trials Japan K.K.
Outcome results
None listed