Amyotrophic Lateral Sclerosis
Conditions
Keywords
Bile acids, Deoxycholic Acid
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
Interventions
Tauroursodeoxycholic acid (TUDCA) 250 mg capsules Doses: 4 capsules (1 g) twice daily 10-15 minutes after a meal Mode of administration: orally Duration: 18 months
Sponsors
University of Ulm
University of Sheffield
University Hospital, Tours
KU Leuven
UMC Utrecht
University of Dublin, Trinity College
Bruschettini S.r.l.
Istituto Superiore di Sanità
Motor Neurone Disease Association
Humanitas Mirasole SpA
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Belgium, France, Germany, Ireland, Italy, Netherlands, United Kingdom
Contacts
Primary ContactAlberto Albanese, MD
Outcome results
None listed