Table 2 of
Daruich, Mol Vis 2019; 25:610-624. Table 2 of
Daruich, Mol Vis 2019; 25:610-624.
Table 2. Clinical studies evaluating bile acids in neurodegenerations.
| Biliary acid | Study design | Number of patients | Disease | Dose | Route | Main results |
|---|---|---|---|---|---|---|
| UDCA | Randomized, non-controlled | 18 | Amyotrophic lateral sclerosis | 15, 30 or 50 mg/kg/day during 4 weeks | Orally | -Excellent safety and tolerability -Cerebrospinal fluid penetration in a dose-dependent manner |
| UDCA | Double-blind, placebo-controlled, randomized, crossover, single center, phase III trial | 63 (16 analyzed) | Amyotrophic lateral sclerosis | 3.5 g/140 ml/day for 3 months | Orally (solubilized) | The rate of progression (assessed by the Appel ALS rating scale) was significantly lower in patients treated with UDCA compared to placebo |
| TUDCA | Double-blind placebo- controlled, randomized, multi-center, phase II trial | 34 (29 analyzed) | Amyotrophic lateral sclerosis | 1 g day for 54 weeks | Orally | Deterioration of function (assed by the ALS Functional Rating Scale Revised) was significantly slower in TUDCA group compared to placebo |