Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS (TUDCA-ALS)

  • STATUS
    Recruiting
  • End date
    Dec 30, 2022
  • participants needed
    440
  • sponsor
    Humanitas Mirasole SpA
Updated on 3 February 2022
dysphagia
pulmonary function test
primary lateral sclerosis
riluzole

Summary

This is a Phase III, multi-centre, randomized, double-blind, placebo-controlled, parallel-group study to evaluate Safety and Efficacy of Tauroursodeoxycholic (TUDCA) as add-on Treatment in Patients Affected by Amyotrophic Lateral Sclerosis (ALS).

Description

Enrolled patients will be randomized to one of two treatment arms: TUDCA or identical placebo by oral route. The randomization will be performed in a ratio one to one for the two arms.

TUDCA will be administered orally at the dose of 1 g twice daily (2 g daily) for 18 months. Patients will be taking also riluzole at the dose of 50 mg twice daily (100 mg daily).

Patient randomization will take place after a screening (lead-in) period of 12 weeks (3 months) with 3 assessments at 6-week intervals. Clinical assessments during the trial phase will be performed every three months. This will allow measuring the progression rate before and after starting treatment (either active or placebo).

Details
Condition Amyotrophic Lateral Sclerosis (ALS)
Treatment Placebo, Tauroursodeoxycholic acid
Clinical Study IdentifierNCT03800524
SponsorHumanitas Mirasole SpA
Last Modified on3 February 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Probable laboratory-supported, probable, or definite ALS, as defined by El Escorial Revised ALS diagnostic criteria 34
Disease duration 18 months
No swallowing difficulty (4 at ALSFRS-R swallowing subscore)
Able to perform reproducible pulmonary function tests
Forced vital capacity 70% of normal
Stable on riluzole treatment for 3 months in the lead-in period
Able to perform reproducible pulmonary function tests
Signed informed consent

Exclusion Criteria

Treatment with edaravone
Other causes of neuromuscular weakness
Presence of other neurodegenerative diseases
Significant cognitive impairment, clinical dementia or psychiatric illness
Severe cardiac or pulmonary disease
Other diseases precluding functional assessments
Other life-threatening diseases
At the time of screening, any use of non-invasive ventilation (e.g. continuous positive airway pressure, non-invasive bi-level positive airway pressure or non-invasive volume ventilation) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
Gastrointestinal disorder that is likely to impair absorption of study drug from the gastrointestinal tract
Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing
Any clinically significant laboratory abnormality
Other concurrent investigational medications
Active peptic ulcer
Previous surgery or infections of small intestine
Patients unable to easily swallow the treatment pills at time of enrolment
Occurrence of frequent biliary colic, biliary infections, severe pancreatic abnormalities
Subjects who weigh 88 lbs (40 kg) or less at screening
Aspartate aminotransferase or alanine aminotransferase concentrations more than 3 times the upper limit of normal
Creatinine clearance 50 ml/min or less
Previous exposure to bile acids
Any clinically significant neurological, haematological, autoimmune, endocrine, cardiovascular, neoplastic, 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 interpretation of study results
Consideration by the investigator, for any reason, that the subject is an unsuitable candidate to receive TUDCA or that the subject is unable or unlikely to comply with the dosing schedule or study evaluations
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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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