Open Label Extension of TUDCA-ALS Study

Phase 3

Recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Tauroursodeoxycholic Acid

• Registration Number: NCT05753852
• Lead Sponsor: Humanitas Mirasole SpA

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

Study Timeline

• Study Start: 2021-10-25
• Status Verified: 2023-02
• Study First Submitted: 2023-02-23
• Study First Submitted QC: 2023-02-23
• Last Update Submitted: 2023-02-23
• Study First Posted: 2023-03-03
• Last Update Posted: 2023-03-03
• Primary Completion: 2025-03-31
• Study Completion: 2025-03-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 184

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Active treatment	Tauroursodeoxycholic Acid	-

Primary Outcome Measures

Name	Time	Method
Long-term safety and tolerability of TUDCA during the open-label phase	18 months	Long-term safety and tolerability assessed through adverse reaction, concomitant treatment, and routine biochemistry analyses

Secondary Outcome Measures

Name	Time	Method
Survival time	18 months	Survival time measured by death or respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ≥22 h per day for ≥10 consecutive days).
Change in disease progression and functional impairment	18 months	Change in disease progression and functional impairment as measured by ALSFRS-R.

Trial Locations

Locations (23)

Katholieke Universiteit Leuven; 🇧🇪 Leuven, Belgium

AOU Città della Salute e della Scienza di Torino; 🇮🇹 Torino, Italy

University of Sheffield; 🇬🇧 Sheffield, United Kingdom

Centre Hospitalier Universitaire de Montpellier; 🇫🇷 Montpellier, France

The Walton Centre NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

Trinity College Dublin; 🇮🇪 Dublin, Ireland

IRCCS Istituto Auxologico Italiano; 🇮🇹 Milano, Italy

Centre Hospitalier Universitaire de Bordeaux; 🇫🇷 Bordeaux, France

Centre Hospitalier Regional Universitaire de Tours; 🇫🇷 Tours, France

Universitätsklinikum Carl Gustav Carus Dresden; 🇩🇪 Dresden, Germany

Centre Hospitalier Universitaire Limoges; 🇫🇷 Limoges, France

NEuroMuscular Omnicentre. Fondazione Serena Onlus; 🇮🇹 Milano, Italy

IRCCS Istituto Clinico Humanitas; 🇮🇹 Rozzano, Italy

Azienda Ospedaliera Santa Maria di Terni; 🇮🇹 Terni, Italy

Plymouth Hospitals NHS Trust; 🇬🇧 Plymouth, United Kingdom

Salford Royal NHS Foundation Trust; 🇬🇧 Salford, United Kingdom

Medizinische Hochschule Hannover; 🇩🇪 Hannover, Germany

Universitätsklinikum Jena; 🇩🇪 Jena, Germany

Universität Ulm; 🇩🇪 Ulm, Germany

AOU Università degli Studi della Campania "Luigi Vanvitelli"; 🇮🇹 Napoli, Italy

Universitair Medisch Centrum Utrecht; 🇳🇱 Utrecht, Netherlands

Royal Stoke University Hospital; 🇬🇧 Stoke, United Kingdom

Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany