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

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Updated on 3 February 2022

See if I qualify

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 Identifier	NCT03800524
Sponsor	Humanitas Mirasole SpA
Last Modified on	3 February 2022

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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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Step 3 Enroll in the clinical study

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Step 4 Get your study results

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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Study Definition

Wikipedia

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