ALS
Conditions
Interventions
Sponsors
Humanitas Mirasole SpA
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. laboratory-supported, probable, or definite ALS diagnosis, as defined by El Escorial Revised ALS diagnostic criteria 2. Disease duration <= 18 months 3. No swallowing difficulty (4 at ALSFRS-R swallowing subscore) 4. Able to perform reproducible pulmonary function tests 5. Forced vital capacity >=70% of normal 6. Stable on riluzole treatment for 3 months in the lead-in period 7. Signed informed consent All patients who entered and completed the TUDCA-ALS clinical trial can be included in the open-label TUDCA-ALS Extension. Inclusion criteria: 1. Completion of the visit M18 (Month 18) of the TUDCA-ALS clinical trial. 2. Signed informed consent for participation in the TUDCA-ALS Extension sub-study.
Exclusion criteria
Exclusion criteria: 1. Treatment with edaravone or other unaccepted concomitant therapy (substances inhibiting the intestinal absorption of bili-ary acids, antacids containing aluminium hydroxide and/or smectites, estrogens and drugs acting by lowering plasmatic cholesterol; drugs increasing biliary clearance of cholesterol, hepatolesive drugs) 2. Other causes of neuromuscular weakness 3. Presence of other neurodegenerative diseases 4. Significant cognitive impairment, clinical dementia or psychiat-ric illness 5. Severe cardiac or pulmonary disease 6. Other diseases precluding functional assessments 7. Other life-threatening diseases 8. At the time of screening, any use of non-invasive ventilation for any portion of the day, or mechanical ventilation via tracheost-omy, or on any form of oxygen supplementation 9. Gastrointestinal disorder that is likely to impair absorption of study drug from the gastrointestinal tract 10. Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing 11. Any clinically significant laboratory abnormality 12. Other concurrent investigational medications 13. Active peptic ulcer 14. Previous surgery or infections of small intestine 15. Patients unable to easily swallow the treatment pills at time of enrolment 16. Occurrence of frequent biliary colic, biliary infections, severe pancreatic abnormalities 17. Subjects who weigh 88 lbs (40 kg) or less at screening 18. Sieric Aspartate aminotransferase or alanine aminotransferase concentrations more than 3 times the upper limit of normal 19. Creatinine clearance 50 ml/min or less 20. Any clinically significant neurological, haematological, autoim-mune, endocrine, cardiovascular, neo-plastic, renal, gastroin-testinal, or other disorder that, in the Investigator's opinion, could interfere with the subject's participation in the study, place the subject at increased risk, or confound interpretation of study results 21. Consideration by the investigator, for any reason, that the sub-ject is an unsuitable candidate to receive TUDCA or that the subject is unable or unlikely to comply with the dosing schedule or study evaluations 22. The patient is sexually active and is not willing to use highly ef-fective contraception during the study and up to 90 days after the day of last dose Exclusion criteria: 1. Treatment with edaravone or other unaccepted concomitant therapy (as per section 8 of the main TUDCA-ALS clinical trial protocol) 2. Consideration by the investigator, for any reason, that the subject is an unsuitable candidate to re-ceive TUDCA or that the subject is unable or unlikely to comply with the dosing schedule or study evaluations 3. The patient of reproductive potential is sexually active and is not willing to use highly effective con-traception during the study and up to 90 days after the day of last dose (see Contraceptive Guidance in Appendix A) 4. The patient is pregnant or breast feeding
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of the study is: * To assess the efficacy of TUDCA in slowing disease progression in patients with ALS during the treatment period compared to the lead-in phase), as measured by ALSFRS-R. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objectives of this study are: * To assess the efficacy of TUDCA in slowing disease progression and functional impairment in patients with ALS, as measured by the survival time, the ALSAQ-40 questionnaire, FVC, the EQ-5D, and muscle force * To assess the long term safety and tolerability of TUDCA for up to 18 months in patients with ALS | — |
Countries
Netherlands
Outcome results
None listed