Amyotrophic lateral sclerosis
Conditions
Interventions
Primidone treatment group:Primidone
Placebo group:Placebo
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 of age (inclusive) at the time of signing the informed consent form. (2) Subjects are diagnosed with possible, clinically probable, clinically probable laboratory-supported, or clinically definite ALS according to the revised El Escorial World Federation of Neurology criteria. (3) The subject's ALS disease duration (from the onset of the first symptom to the screening visit) is =2 years. (4) The pre-study ALSFRS-R slope of the subject prior to the screening visit must be =0.5 points/month (?ALSFRS-R = (48 - ALSFRS-R score at the screening visit) / duration in months between the screening visit and disease onset). (5) Forced Vital Capacity (FVC) must be =50% of the predicted value. (6) At the screening visit, the subject must be capable of swallowing the study tablets. (7) The subject is not currently receiving riluzole treatment, or has been on a stable dose of riluzole for at least 4 weeks prior to the screening visit. Subjects on riluzole treatment are expected to maintain the same dose throughout the study period. (8) The subject must not be currently receiving edaravone treatment, or must be undergoing treatment with an approved standard regimen of edaravone. 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 period. (9) Subjects with a body weight not less than 45 kg and a Body Mass Index (BMI) not less than 18.0 kg/m². (10) The subject has signed the informed consent form and is able to comply with the requirements and restrictions listed in this protocol.
Exclusion criteria
Exclusion criteria: (1) Subjects with a history of convulsive seizures 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), or substance abuse (including drugs, alcohol, tobacco, and caffeine). (3) Subjects with severe infection (e.g., infectious pneumonia, sepsis) within 4 weeks prior to the screening visit; infection 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's judgment. (4) Subjects with active herpes zoster infection within 2 months prior to the screening visit. (5) Subjects with a documented suicide attempt within 6 months prior to the screening visit, or those judged by the investigator to be at risk of suicide attempt. (6) Subjects with a history of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease, or any other significant medical condition besides ALS that would preclude their safe participation in this study. (7) Subjects who are pregnant or breastfeeding. (8) Subjects with a known history of allergy to primidone. (9) Subjects who have received live vaccines within 14 days prior to the screening visit. (10) Subjects concurrently participating in any other interventional clinical trial or having received another investigational drug within 4 weeks prior to the screening visit or within 5 half-lives of the study drug (whichever is longer). (11) Subjects who have received stem cell or gene therapy for ALS at any time in the past. (12) Subjects who test positive for Human Immunodeficiency Virus (HIV) antibodies. (13) Subjects with abnormal laboratory findings at the screening visit, including: Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) > 3.0 x Upper Limit of Normal (ULN); Bilirubin > 1.5 x ULN; Serum Albumin < 3.5 g/dL; Estimated Glomerular Filtration Rate (eGFR) < 60 mL/min/1.73 m²; or other abnormal laboratory values considered clinically significant by the investigator. (14) Subjects with a positive test result for Hepatitis B surface antigen (HBsAg) or anti-Hepatitis B core antibody (anti-HBcAb) at the screening visit or within 3 months prior to the first dose of the study drug. (15) Subjects with a positive test result for Hepatitis C antibodies at the screening visit or within 3 months prior to the initiation of the study drug. (16) Subjects who have taken the investigational drug (primidone) within 1 month prior to the screening visit, or test positive for blood concentrations of phenobarbital, an intermediate metabolite of primidone. (17) Subjects who are unable to comply with the study protocol for taking primidone.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ALSRFS-R Scale Score;Blood IL-8 levels; | — |
Secondary
| Measure | Time frame |
|---|---|
| muscle strength;ALSAQ-5 Scale Score;CNS-BFS Scale Score;HAMD Scale Score;SDS Scale Score;Blood NfL levels;Blood RIPK1 levels; | — |
Countries
China
Contacts
Public ContactJun Wei
Yichang Central People's Hospital
Outcome results
None listed