Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis
Status:
Completed
Trial end date:
2012-04-01
Target enrollment:
Participant gender:
Summary
The preclinical rationale for tauroursodeoxycholic acid (TUDCA) use in treating patients with
amyotrophic lateral sclerosis (ALS) stems from the demonstration of antioxidant,
antiapoptotic and neuroprotective properties of TUDCA in the central nervous system (CNS),
both in vitro and in vivo models.
This protocol is meant for assessing if the addition of TUDCA to the conventional therapy can
improve the therapeutic outcome in patients affected by ALS.
Safety will be assessed for all subjects, for the entire duration of the study. 30 patients
affected by ALS with site of onset in the limbs will be recruited.
All enrolled subjects will continue receiving riluzole at the same regimen as before entering
the trial. Based on an appropriate random code, subjects will be divided into two groups of
equal size treated, after a lead-in period of 3 months, by oral route with TUDCA at the dose
2 g daily for 1 year or with identical placebo by oral route at the same dosing schedule,
under double-blind conditions.
Every concomitant and/or supportive therapy will be admitted.
Evaluation criteria:
Efficacy. The proportion of responder patients in the two treatment groups was the primary
outcome measure of the study. Responder patients were defined as those subjects showing an
improvement of at least 15% in the ALSFRS-R (2) slope during the treatment period as compared
to the lead-in period. This threshold was chosen based according to the consensus conference
on designing and implementing clinical trials in ALS (3).
Other parameters will include ALSFRS-R at study end, FVC%, the SF-36 quality of life rating
scale, time to tracheotomy from starting of study medication dosing (if appropriate),
survival Time from starting of study medication dosing (if appropriate), Medical Research
Council scores for right and left muscle groups.
Safety. Incidence, severity and type of adverse events; changes in clinical laboratory
findings.
Phase:
Phase 2
Details
Lead Sponsor:
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta