Safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS)

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 440

Inclusion Criteria

1. Probable laboratory-supported, probable, or definite ALS, as defined by El Escorial Revised ALS diag-nostic criteria 38
2. Disease duration = 18 months
3. No swallowing difficulty (4 at ALSFRS-R swallowing subscore)
4. Able to perform reproducible pulmonary function tests
5. Forced vital capacity =70% of normal
6. Stable on riluzole treatment for 3 months in the lead-in period
7. Signed informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 300
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 140

Exclusion Criteria

8. Treatment with edaravone or other unaccepted concomitant therapy (as per Section 8)
9. Other causes of neuromuscular weakness
10. Presence of other neurodegenerative diseases
11. Significant cognitive impairment, clinical dementia or psychiatric illness
12. Severe cardiac or pulmonary disease
13. Other diseases precluding functional assessments
14. Other life-threatening diseases
15. At the time of screening, any use of non-invasive ventilation (e.g. continuous positive airway pres-sure, non-invasive bi-level positive airway pressure or non-invasive volume ventilation) for any por-tion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementa-tion
16. Gastrointestinal disorder that is likely to impair absorption of study drug from the gastrointestinal tract
17. Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing
18. Any clinically significant laboratory abnormality
19. Other concurrent investigational medications
20. Active peptic ulcer
21. Previous surgery or infections of small intestine
22. Patients unable to easily swallow the treatment pills at time of enrolment
23. Occurrence of frequent biliary colic, biliary infections, severe pancreatic abnormalities
24. Subjects who weigh 88 lbs (40 kg) or less at screening
25. Aspartate aminotransferase or alanine aminotransferase concentrations more than 3 times the up-per limit of normal
26. Creatinine clearance 50 ml/min or less
27. Any clinically significant neurological, haematological, autoimmune, endocrine, cardiovascular, neo-plastic, renal, gastrointestinal, or other disorder that, in the Investigator's opinion, could interfere with the subject's participation in the study, place the subject at increased risk, or confound inter-pretation of study results
28. Consideration by the investigator, for any reason, that the subject is an unsuitable candidate to re-ceive TUDCA or that the subject is unable or unlikely to comply with the dosing schedule or study evaluations
29. The patient is sexually active and is not willing to use highly effective contraception during the study and up to 90 days after the day of last dose (see Contraceptive Guidance in Appendix A).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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