Clinical trial NCT05753852: Open Label Extension of TUDCA-ALS Study
Official title: Open Label Extension Study to Investigate Long Term Safety, Tolerability and Efficacy of Tauroursodeoxycholic Acid in Patients With ALS Who Completed the TUDCA-ALS Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Bile acids
Deoxycholic Acid
Lead sponsor: Humanitas Mirasole SpA
Sponsor class: OTHER
Collaborators: name: University of Ulm
name: University of Sheffield
name: University Hospital, Tours
name: KU Leuven
name: UMC Utrecht
name: University of Dublin, Trinity College
name: Bruschettini S.r.l.
name: Istituto Superiore di Sanità
name: Motor Neurone Disease Association
First posted: 2023-03-03
Enrollment: 184
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will provide extended access to patients and assess longer-term outcomes on patients who have completed the TUDCA-ALS study.

Detailed description: The TUDCA-ALS Open label extension study is designed to investigate long term safety, tolerability and efficacy of tauroursodeoxycholic acid in patients with ALS who completed the TUDCA-ALS study

Eligibility criteria: Inclusion Criteria:

* Completion of the visit M18 (Month 18) of the TUDCA-ALS clinical trial.
* Signed informed consent for participation in the TUDCA-ALS Extension sub-study

Exclusion Criteria:

* Treatment with edaravone or other unaccepted concomitant therapy
* 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 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
* The patient is pregnant or breast feeding

Primary outcomes:
measure: Long-term safety and tolerability of TUDCA during the open-label phase
timeFrame: 18 months
Secondary outcomes:
measure: Survival time
timeFrame: 18 months
measure: Change in disease progression and functional impairment
timeFrame: 18 months
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: Katholieke Universiteit Leuven
city: Bordeaux
country: France
facility: Centre Hospitalier Universitaire de Bordeaux
city: Limoges
country: France
facility: Centre Hospitalier Universitaire Limoges
city: Montpellier
country: France
facility: Centre Hospitalier Universitaire de Montpellier
city: Tours
country: France
facility: Centre Hospitalier Regional Universitaire de Tours
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus Dresden
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Jena
country: Germany
facility: Universitätsklinikum Jena
city: Ulm
country: Germany
facility: Universität Ulm
city: Dublin
country: Ireland
facility: Trinity College Dublin
city: Milano
country: Italy
facility: IRCCS Istituto Auxologico Italiano
city: Milano
country: Italy
facility: NEuroMuscular Omnicentre. Fondazione Serena Onlus
city: Napoli
country: Italy
facility: AOU Università degli Studi della Campania "Luigi Vanvitelli"
city: Rozzano
country: Italy
facility: IRCCS Istituto Clinico Humanitas
city: Terni
country: Italy
facility: Azienda Ospedaliera Santa Maria di Terni
city: Torino
country: Italy
facility: AOU Città della Salute e della Scienza di Torino
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Liverpool
country: United Kingdom
facility: The Walton Centre NHS Foundation Trust
city: Plymouth
country: United Kingdom
facility: Plymouth Hospitals NHS Trust
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
city: Sheffield
country: United Kingdom
facility: University of Sheffield
city: Stoke
country: United Kingdom
facility: Royal Stoke University Hospital
Study officials:
References: