A randomized double-blind clinical trial on safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS): the statistical analysis plan of TUDCA-ALS trial.

Publication date: Dec 05, 2023

Amyotrophic lateral sclerosis (ALS) is a highly debilitating neurodegenerative condition. Despite recent advancements in understanding the molecular mechanisms underlying ALS, there have been no significant improvements in therapeutic options for ALS patients in recent years. Currently, there is no cure for ALS, and the only approved treatment in Europe is riluzole, which has been shown to slow the disease progression and prolong survival by approximately 3 months. Recently, tauroursodeoxycholic acid (TUDCA) has emerged as a promising and effective treatment for neurodegenerative diseases due to its neuroprotective activities. The ongoing TUDCA-ALS study is a double-blinded, parallel arms, placebo-controlled, randomized multicenter phase III trial with the aim to assess the efficacy and safety of TUDCA as add-on therapy to riluzole in patients with ALS. The primary outcome measure is the treatment response defined as a minimum of 20% improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R) slope during the randomized treatment period (18 months) compared to the lead-in period (3 months). Randomization will be stratified by country. Primary analysis will be conducted based on the intention-to-treat principle through an unadjusted logistic regression model. Patient recruitment commenced on February 22, 2019, and was closed on December 23, 2021. The database will be locked in September 2023. This paper provides a comprehensive description of the statistical analysis plan in order to ensure the reproducibility of the analysis and avoid selective reporting of outcomes and data-driven analysis. Sensitivity analyses have been included in the protocol to assess the impact of intercurrent events related to the coronavirus disease 2019. By focusing on clinically meaningful and robust outcomes, this trial aims to determine whether TUDCA can be effective in slowing the disease progression in patients with ALS. ClinicalTrials. gov NCT03800524 . Registered on January 11, 2019.

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Concepts Keywords
Als Amyotrophic lateral sclerosis
Clinicaltrials Bile acids
Coronavirus Clinical trial
February Double-blind
Phase III
Randomized
Statistical analysis plan

Semantics

Type Source Name
drug DRUGBANK Tauroursodeoxycholic acid
disease MESH amyotrophic lateral sclerosis
pathway KEGG Amyotrophic lateral sclerosis
drug DRUGBANK Riluzole
disease MESH disease progression
disease VO effective
disease MESH neurodegenerative diseases
disease IDO country
disease VO protocol
disease MESH coronavirus disease 2019
disease MESH lateral sclerosis
disease VO Iss
drug DRUGBANK Cholic Acid
drug DRUGBANK Trestolone
disease VO time
drug DRUGBANK Methionine
disease VO document
drug DRUGBANK Ademetionine
disease VO oral route
disease VO dose
disease VO Pla
drug DRUGBANK Isoxaflutole
disease MESH death
drug DRUGBANK Nevirapine
disease VO population
disease IDO site
disease MESH psychiatric illness
disease MESH peptic ulcer
disease MESH infections
disease MESH respiratory insufficiency
disease IDO blood
disease IDO history
disease MESH clinical importance
drug DRUGBANK Tropicamide
disease VO report
drug DRUGBANK Creatinine
drug DRUGBANK Phosphocreatine
disease IDO symptom
disease VO monthly
disease VO vaccination
disease IDO infection
disease MESH complications
disease VO adverse event
disease MESH rare diseases
disease VO company
drug DRUGBANK Diethylstilbestrol
drug DRUGBANK Edaravone
disease MESH AIDS

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