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. 2020 Jul 7;11:614. doi: 10.3389/fneur.2020.00614

Table 2.

Clinical studies on trampirosate.

Study Study design Number of patients Disease Treatment Results
Aisen et al. (41) RCT
n = 58
Mild-to-moderate AD Placebo, 3-APS 50, 100, or 150 mg bid during 3 months 3-APS reduced CSF Aβ42 levels after 3 months of treatment
Alphase Study.Aisen et al. (42) RCT
n = 1,052
Mild-to-moderate AD Placebo, tramiprosate 100 mg or tramiprosate 150 mg bid during 78 weeks No significant treatment effect.Trend toward a treatment effect for ADAS-cog and less HV loss.
Abushakra et al. (29) RCT
n = 2,025 (pooled data from two phase III studies).
Three subgroups of patients with mild-to-moderate AD:ApoE4 homozygotes, ApoE4 heterozygotes and non-carriers Placebo, tramiprosate 100 mg or tramiprosate 150 mg bid during 78 weeks In ApoE4/4 homozygotes on 150 mg bid significant effects on ADAS-cog and positive trends on CDR-SB.Intermediate efficacy in ApoE4 heterozygotes and no benefit in non-carriers.
Abushakra et al. (43) RCT
n = 2,025 (pooled data from two phase III studies).
Three subgroups of patients with mild-to-moderate AD:ApoE4 homozygotes, ApoE4 heterozygotes and non-carriers Placebo or 100 mg bid, or 150 mg tramiprosate bid Highest efficacy (ADAS-cog, CDR-SB, and DAD scores) in ApoE4/4 homozygotes on 150 mg bid with mild disease.The mild subgroup showed cognitive stabilization with no decline over 78 weeks, both ADAS-cog and DAD effects increased over time.
Sabbagh et al. (46) RCT
A substudy of 257 subjects of Alphase Study.
Mild-to-moderate AD patients ApoE4 homozygotes Placebo or 100 mg bid, or 150 mg tramiprosate bid Statistically significant efficacy in the mild subgroup (ADAS and CDR scores).Some benefit at week 78 as indicated by DAD scores.
Spalletta et al. (33) Controled study
n = 33
aMCI Tramiprosate 50 mg qd for 2 weeks and bid for the next year vs. untreated patients Decreased volume loss in the left and right hippocampal tail, left and right fusiform gyrus, and right inferior temporal cortex.Improved short-term episodic memory performance.
Bossù et al. (32) Open trial
n = 20
aMCI Tramiprosate 50 mg qd for 2 weeks and bid for the next year Compared to baseline, patients with ApoE4 allele showed a significant decrease in IL-18 and improved short-term episodic memory performance.
Martorana et al. (50) Observational retrospective study
n = 245
aMCI and naMCI Tramiprosate 100 mg qd Significant improvements in cognitive decline (MMSE score) in patients with aMCI (at months 8 and 12), and naMCI (at month 4)
Martorana et al. (51) Open trial
n = 10
aMCI 100 mg tramiprosate during 4 weeks Measurable changes of SLAI in aMCI patients. Potential role in enhancing cholinergic transmission by modulating the inhibitory cortical activity.
Gauthier et al. (44) RCT
Subset of 312 Alphase Study subjects.
Mild-to-moderate AD patients Placebo, tramiprosate 100 mg or tramiprosate 150 mg bid during 78 weeks Hippocampal atrophy slowing, beneficial effect on cognition (ADAS-cog)
Saumier et al. (45) RCT
n = 1,052 (Alphase study data)
Mild-to-moderate AD patients Placebo, tramiprosate 100 mg or tramiprosate 150 mg bid during 18 months ADAS-cog change scores and HV change correlate with vMRI.
Ricciardi et al. (52) RCT
n = 47
Parkinson disease Tramiprosate 100 mg bid vs. untreated patients Benefits on non-motor symptoms (UPDRS-I) and beneficial effect on excessive sleepiness (Epworth Sleepiness Scale).
Greenberg et al. (53) RCT
n = 24
Lobar intracerebral hemorrhage, with possible or probableCAA Tramiprosate 50, 100, or 150 mg bid during 12 weeks No significant changes on neurological function (NIHSS), daily functioning (Barthel Index), cognition (ADAS-cog), and executive function (EXIT25).

3-APS, 3-amino-1-propanesulfonic acid; AD, Alzheimer's disease; ADAS, Alzheimer's Disease Assessment Scale; ADAS-cog, Alzheimer Disease Assessment Scale-cognitive subscale; aMCI, amnestic MCI; ApoE4, Apolipoprotein E4; ε4 allele of the apolipoprotein E gene; bid, twice daily; CAA, cerebral amyloid angiopathy; CDR-SB, Clinical Dementia Rating Scale, sum of boxes; DAD, Disability Assessment for Dementia; HV, hippocampus volume; MCI, Mild Cognitive Impairment; MMSE, Mini Mental State Examination; naMCI, non-amnestic MCI; NIHSS, NIH Stroke Scale; qd, daily; RCT, randomized controlled trial; SLAI, short latency afferent inhibition; UPDRS, Unified Parkinson's Disease Rating Scale; vMRI, volumetric magnetic resonance imaging.