Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS (TUDCA-ALS)
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|ClinicalTrials.gov Identifier: NCT03800524|
Recruitment Status : Active, not recruiting
First Posted : January 11, 2019
Last Update Posted : July 10, 2023
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis (ALS)||Drug: Tauroursodeoxycholic Acid Drug: Placebo||Phase 3|
Enrolled patients will be randomized to one of two treatment arms: TUDCA or identical placebo by oral route. The randomization will be performed in a ratio one to one for the two arms.
TUDCA will be administered orally at the dose of 1 g twice daily (2 g daily) for 18 months. Patients will be taking also riluzole at the dose of 50 mg twice daily (100 mg daily).
Patient randomization will take place after a screening (lead-in) period of 12 weeks (3 months) with 3 assessments at 6-week intervals. Clinical assessments during the trial phase will be performed every three months. This will allow measuring the progression rate before and after starting treatment (either active or placebo).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||337 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Safety and Efficacy of Tauroursodeoxycholic (TUDCA) as add-on Treatment in Patients Affected by Amyotrophic Lateral Sclerosis (ALS)|
|Actual Study Start Date :||February 22, 2019|
|Estimated Primary Completion Date :||December 31, 2023|
|Estimated Study Completion Date :||December 31, 2023|
Experimental: Tauroursodeoxycholic acid (TUDCA)
Drug: Tauroursodeoxycholic Acid
Placebo Comparator: Reference therapy
- Number of responder patients [ Time Frame: 18 months ]Identification of the responder patients defined as those showing an improvement of at least 20% in the ALSFRS-R slope
- Survival time [ Time Frame: 18 months ]Survival time measured by death or respiratory insufficiency
- ALS disease functional rating scale - revised version (ALSFRS-R) [ Time Frame: 18 months ]Difference in change from baseline in ALSFRS-R. Each task of the scale is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score ranging from 0 = worst to 48 = best.
- ALS Assessment Questionnaire-40 (ALSAQ-40) [ Time Frame: 18 months ]Difference in change from baseline in ALSAQ-40. The instrument contains 40 statements that measure five dimensions of health state: Physical Mobility (10 statements), Activities of Daily Living and Independence (10 statements), Eating and Drinking (5 statements), Communication (5 statements), Emotional Functioning (10 statements). The patient must indicate how often (Never, Rarely, Sometimes, Often, or Always) the statement have been true. Dimension scores are coded on a likert scale, ranging from 0 (best health as assessed by the scale) to 100 (worse health as assessed by the measure).
- Forced Vital Capacity [ Time Frame: 18 months ]Difference in change from baseline in Forced Vital Capacity
- EuroQol 5-Dimension-5 Levels (EQ-5D-5L) scale [ Time Frame: 18 months ]Difference in change from baseline in EQ-5D-5L scale. The EQ-5D-5L descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 5 levels: 1.no problems, 2.slight problems, 3.moderate problems, 4.severe problems, 5.extreme problems. The patient is asked to indicate his/her health state by ticking (or placing a cross) in the box against the most appropriate statement in each of the 5 dimensions. This decision results in a 1-digit number expressing the level selected for that dimension. The digits for 5 dimensions can be combined in a 5-digit number describing the patient health state. Numbers 1-5 have no arithmetic properties and should not be used as a cardinal score.
- Medical Research Council (MRC) scale [ Time Frame: 18 months ]Difference in change from baseline in muscle force assessed by the MRC scale. The scale rates muscle strength of 6 muscles (3 at the upper and 3 at the lower limbs), bilaterally. Each muscle is graded from 0 = no movement, to 5 = normal strength, giving a total sum-score that ranges from 0 (total paralysis) to 60 (normal strength).
- Neurofilaments levels [ Time Frame: 18 months ]Effect of TUDCA on Neurofilament levels in comparison to placebo
- MMP-9 levels [ Time Frame: 18 months ]Effect of TUDCA on MMP-9 expression in comparison to placebo
- Long-term safety and tolerability [ Time Frame: 18 months ]assessed through adverse reaction, concomitant treatment, physical examination, vital signs and routine haematology and biochemistry analyses
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03800524
|Study Chair:||Alberto Albanese, MD||Humanitas Mirasole SpA|