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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 320

Inclusion Criteria

1.laboratory-supported, probable, or definite ALS diagnosis, as defined by El Escorial Revised ALS diagnostic criteria at screen-ing visit (M-3)
2.Disease duration = 18 months at screening visit (M-3)
3.Able to perform reproducible pulmonary function tests at screening visit (M-3)
4.FVC or SVC =70% of normal at screening visit (M-3)
5.Stable on riluzole treatment for 3 months in the lead-in period
6.Signed informed consent at screening visit (M-3)

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 250
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 70

Exclusion Criteria

1.Treatment with edaravone or other unaccepted concomitant therapy (substances inhibiting the intestinal absorption of bili-ary acids, antacids containing aluminium hydroxide and/or smectites, estrogens and drugs acting by lowering plasmatic cholesterol; drugs increasing biliary clearance of cholesterol, hepatolesive drugs)
2.Other causes of neuromuscular weakness
3.Presence of other neurodegenerative diseases
4.Significant cognitive impairment, clinical dementia or psychiat-ric illness
5.Severe cardiac or pulmonary disease
6.Other diseases precluding functional assessments
7.Other life-threatening diseases
8.Any use of non-invasive ventilation for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
9.Gastrointestinal disorder that is likely to impair absorption of study drug from the gastrointestinal tract
10.Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dos-ing
11.Any clinically significant laboratory abnormality
12.Other concurrent investigational medications
13.Active peptic ulcer
14.Previous surgery or infections of small intestine
15.Patients unable to easily swallow the treatment pills
16.Acute inflammation of the gallbladder or bile ducts
17.Occurrence of frequent biliary colic, biliary infections, severe pancreatic abnormalities
18.Bile duct obstruction, calcified X-ray opaque gallstones and re-duced mobility of the gallbladder
19.Subjects who weigh 88 lbs (40 kg) or less
20.Sieric Aspartate aminotransferase or alanine aminotransferase concentrations more than 3 times the upper limit of normal
21.Creatinine clearance 50 ml/min or less
22.Any clinically significant neurological, haematological, autoim-mune, endocrine, cardiovascular, neo-plastic, renal, gastroin-testinal, 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 interpretation of study results
23.Consideration by the investigator, for any reason, that the sub-ject is an unsuitable candidate to receive TUDCA or that the subject is unable or unlikely to comply with the dosing sched-ule or study evaluations
24.The patient of reproductive potential 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
25.The patient is pregnant or breast feeding

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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