Amyotrophic Lateral Sclerosis, ALS
Conditions
Interventions
Experimental group:Part A (Initial Treatment Period, 24 weeks):
Medication: Pumitone tablets
Dosage: Start with a dose gradually increased over 1-2 weeks to the target dose of 50 mg, once every night. If not tolerated, reduce to 25 mg, once every night
Maintenance Dose: All patients ultimately receive an individualized dose treatment of 25 mg–50 mg daily
Part B (Long-term Extension Period, Weeks 24 to 76): Medication and Dosage: Continue to take Pumitone 25 mg–50 mg, once every night.
Sponsors
Yichang Central People’s Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1. Subjects must be between 18 and 80 years old (inclusive) at the time of signing the informed consent form; 2. Subjects must be diagnosed with possible ALS, clinically probable ALS, clinically probable laboratory-supported ALS, or clinically definite ALS according to the revised El Escorial World Federation of Neurology criteria; 3. At the screening visit, subjects must be able to swallow tablets orally or via a nasogastric tube or gastrostomy tube; 4. Subjects must not currently be receiving riluzole treatment, or if they are, they must have been on a stable dose of riluzole for at least 4 weeks prior to the screening visit. Subjects expected to receive riluzole treatment are to maintain the same dose throughout the study; 5. Subjects must not currently be receiving edaravone treatment, or if they are, they must be on an approved standard edaravone regimen. Subjects receiving edaravone treatment must have completed at least 2 cycles prior to the screening visit and are expected to continue edaravone treatment throughout the study; 6. Subjects must weigh at least 45 kg and have a body mass index (BMI) of at least 18.0 kg/m^2; 7. Subjects must sign the informed consent form and be able to comply with the requirements and restrictions outlined in this protocol.
Exclusion criteria
Exclusion criteria: 1. Subjects with a history of convulsions or epilepsy (excluding a history of febrile seizures in childhood); 2. Subjects with significant cognitive impairment, psychiatric disorders, other neurodegenerative diseases (e.g., Parkinson's disease or Alzheimer's disease), neuroimmunological disorders, or substance abuse (including misuse of drugs, alcohol, tobacco, and caffeine); 3. Subjects with a recent severe infection within 4 weeks prior to the screening visit (e.g., infectious pneumonia, sepsis); infections requiring hospitalization or intravenous antibiotic, antiviral, or antifungal treatment within 4 weeks prior to screening; or a history of chronic bacterial infection (e.g., tuberculosis) deemed unacceptable by the investigator; 4. Subjects with active shingles infection within 2 months prior to the screening visit; 5. Subjects with a record of suicide attempt within 6 months prior to the screening visit, or those deemed by the investigator to be at risk of suicide; 6. Subjects with a history of unstable or severe heart, lung, cancer, liver, or kidney diseases, or other major medical conditions (except ALS) that would prevent safe participation in the study; 7. Subjects who are pregnant or breastfeeding; 8. Subjects known to be allergic to PXT; 9. Subjects who received a live vaccine within 14 days prior to the screening visit; 10. Subjects participating in any other interventional clinical trial or who received another investigational drug within 4 weeks prior to the screening visit; 11. Subjects who have ever received stem cell or gene therapy for ALS; 12. Subjects who are HIV antibody positive; 13. Subjects with abnormal laboratory results at the screening visit, including: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3.0× the upper limit of normal (ULN); bilirubin > 1.5× ULN; serum albumin < 3.5 g/dL; estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m^2, or other abnormal lab values considered clinically significant by the investigator; 14. Subjects with positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (anti-HBc) results within 3 months prior to screening or before the first study drug administration; 15. Subjects with positive hepatitis C antibody results within 3 months prior to screening or before the first study drug administration; 16. Subjects who took the investigational drug (PXT) or had positive blood levels of PXT's intermediate metabolite phenobarbital within 1 month prior to screening; 17. Subjects unable to comply with the study protocol for taking PXT.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Changes in the progression rate of ALSFRS-R score compared to baseline.; | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety indicators;Changes in various aspects of the illness; | — |
Countries
China
Contacts
Public ContactJun Wei
Yichang Central People’s Hospital
Outcome results
None listed