Table 3.
Clinical trials of MSA.
| Intervention | Status | ClinicalTrials.gov identifier | Study design | No. of participants | Main outcome measure | Results | |
|---|---|---|---|---|---|---|---|
| Completed | r-hGH (recombinant human growth hormone) (Holmberg et al., 2007) | Compl. | Phase II randomized, placebo-controlled | 22 treated, 21 placebo | Safety, UPDRS, UMSARS, CAFTs | Trend to less worsening in UPDRS, UMSARS and CAFTs suggesting possibility of disease modification; higher dose and/or larger study group (>90) trials recommended | |
| Riluzole (NNIPPS) (Bensimon et al., 2009) | Compl. | NCT00211224 | Phase II/III; randomized, placebo-controlled | 194 treated, 197 placebo | Survival, mortality, different scales (e.g. UPDRS, NNIPPS-PPS) | No evidence of beneficial effect on survival and scales | |
| Minocycline (MEMSA) (Dodel et al., 2010) | Compl. | NCT00146809 | Phase II randomized, placebo-controlled | 31 treated, 31 placebo | UMSARS, UPDRS | No significant difference between treatment groups | |
| Rasagiline (Poewe et al., 2012) | Compl. | NCT00977665 | Phase II; randomized, placebo-controlled | 84 treated, 90 placebo | UMSARS DWI | No significant difference between treatment groups on the primary and secondary endpoints | |
| Lithium carbonate (Sacca et al., 2013) | Compl. | NCT00997672 | Phase II randomized, placebo-controlled | 4 treated, 5 placebo | Safety, UMSARS, micro- and macrostructural MR parameters | Terminated because of side effects | |
| IVIG (intravenous immunoglobulin) (Novak et al., 2012) | Compl. | NCT00750867 | Phase II Open label | 7 treated | Safety, efficacy | Safe, improved UMSARS scores | |
| Autologous mesenchymal stem cells (Lee et al., 2008b) | Compl. | Not listed | Phase I/II Open label | 11 treated, 18 historical control group | Safety, UMSARS, FDG-PET | Safe, improved UMSARS scores compared to controls, increased FDG-uptake in cerebellum and white matter of the frontal cortex in the in the MSC-treated group compared to baseline Increased gray matter density in cerebellum |
|
| Autologous mesenchymal stem cells (Lee et al., 2012) | Compl. | NCT00911365 | Phase II; randomized placebo-controlled | 14 treated, 17 placebo | UMSARS | Delayed progression in UMSARS I and II scores, less extensive decrease in glucose metabolism and gray matter density | |
| Pending | Fluoxetine | Compl. | NCT01146548 | Phase II randomized, placebo-controlled | UMSARS | Pending | |
| Rifampicin | Ongoing study | NCT01287221 | Phase II/III; randomized, placebo-controlled | UMSARS | Pending | ||
RCT, randomized controlled trial; FDG-PET 0, fluorodeoxyglucose PET; MSC, mesenchymal stem cells; PPS, Parkinson-Plus-Scale; CAFTs, cardiovascular autonomic function tests.