Amyotrophic lateral sclerosis
Conditions
Interventions
Experimental group:Sodium phenylbutyrate granules + tauroursodeoxycholic acid capsules
Control group:Sodium phenylbutyrate placebo + tauroursodeoxycholic acid placebo
Sponsors
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1.Aged 18 to 80 years (including the critical value), regardless of gender; 2.The diagnosis meets the diagnostic criteria of El Escorial amyotrophic lateral sclerosis revised by the World Federation of Neurology, which is diagnosed or likely to be sporadic or familial ALS; 3.Less than or equal to 18 months after the onset of symptoms of amyotrophic lateral sclerosis; 4.Clearly understand and voluntarily participate in the study and sign the informed consent form; 5.The forced vital capacity (FVC) in the screening period is greater than or equal to 80% of the estimated vital capacity; 6.Subjects (including male subjects) are willing to have no family planning during the whole study period and within 3 months after the end of the study, voluntarily take effective contraceptive measures, and have no sperm and egg donation plans.
Exclusion criteria
Exclusion criteria: 1.Patients with tracheostomy; 2.Have used PBTUDCA or UDCA within 3 months before screening or plan to use these drugs during the study (except test drugs); 3.Known allergy to PB or bile salt; 4.Abnormal liver function, ALT or AST > 3ULN; 5.Renal insufficiency creatinine > 1.5ULN; 6.Poor control of hypertension (SBP > 160mmhg or DBP > 100mmhg at rest); 7.Pregnant or lactating subjects; 8.History of cholecystectomy; 9.Biliary diseases that hinder bile flow, such as active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, obstructive suppurative cholangitis, gallbladder cancer, gallbladder gangrene, gallbladder abscess, cholelithiasis. 10.Heart failure history of grade III-IV (NYHA grade); 11.Severe pancreatic or gastrointestinal diseases may change the intestinal hepatic circulation and absorption of TUDCA, such as active peptic ulcer, pancreatitis and intestinal obstruction; 12.History of small bowel resection; 13.According to the judgment of the researcher, there are diseases that weaken the subject's ability to provide informed consent, such as unstable mental diseases, cognitive impairment, dementia or drug abuse; 14.Implantation of diaphragm pacemaker; 15.Patients permanently and continuously dependent on ventilator assisted ventilation; 16.The patient who cannot swallow autonomously or the investigator determines that the patient has difficulty swallowing; 17.Have participated in other drug clinical trials or are participating in other clinical trials within 90 days before screening; 18.Any reason that the researcher believes will expose the subject to greater risk or prevent the subject from complying with or completing the study.
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Forced vital capacity;Patient survival;Duration of permanent assisted ventilation;Tracheostomy timing;Time to death;Safety; | — |
Primary
| Measure | Time frame |
|---|---|
| Rate of decline in the amyotrophic lateral sclerosis functional rating scale; | — |
Countries
China
Contacts
Public ContactCui Liying
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Outcome results
None listed