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. 2023 Feb 7;13(2):309. doi: 10.3390/biom13020309

Table 3.

Clinical Profile of 5-HT6 Antagonists Recently in Development.

Agents Indication NCT Number Endpoint Outcome
Avisetron Schizophrenia
(Pilot study) [68,69]
Not available Key Endpoints: PANSS, CAT As augmentation therapy, avisetron treatment-related benefits were observed in PANSS total scores, PANSS positive subscores and CAT
Schizophrenia
(Phase 2 study) [70]
Not available Primary: Change from baseline in the PANSS total scores
Other Key Endpoints: Change from baseline in the CGI-S, CGI-I, NSA-16, PSPS, CogFu, BACS and CPT
As augmentation therapy, a trend towards avisetron treatment-related benefits were observed in PANSS total score, PANSS positive subscores and PANSS general psychopathology scale after 6 weeks of treatment; No notable effects on CGI-S, CGI-I, NSA, PSPS, CogFu, BACS and CPT
Cerlapirdine Alzheimer’s disease
(Pilot study) [71]
NCT00481520 Key Endpoints: MMSE, ADAS-Cog and CANTAB Trend towards improvement was observed with cerlapirdine treatment on the ADAS-Cog 11 and CANTAB at the end of 4 weeks
Alzheimer’s disease
(Phase 2 study) [72]
NCT00895895 Primary: Change from baseline in the ADAS-Cog 11 total scores
Other Key Endpoints: Change from baseline in the ADCS-CGIC, CANTAB and NPI-12
No beneficial effects of cerlapirdine were observed at the end of 24 weeks on any of the studied endpoints
Idalopirdine Schizophrenia
(Pilot study) [73]
Not available Safety, tolerability, pharmacokinetics and pharmacodynamics (cognitive changes assessed using BACS) Safe and well-tolerated as standalone treatment for 14 days; Idalopirdine treatment was associated with dose-dependent pattern of improvement in the BACS endpoint; No effect in the placebo treated group
Schizophrenia
(Phase 2 study) [74]
NCT00810667 Primary: Change in PANSS total scores
Other Key Endpoints: Neurocognitive performance using the BACS
As augmentation therapy, no change was observed in the PANSS total scores or BACS scores or PANSS cognitive subscale scores as compared to placebo after 12 weeks of treatment
Alzheimer’s disease (Phase 2 study) [75] NCT01019421 Primary: Change from baseline in the ADAS-Cog 11
Other Key Endpoints: Change from baseline in the ADCS-ADL, ADCS-CGIC, MMSE and NPI-12
As augmentation therapy, significant improvements in ADAS-Cog 11 scores were observed as compared to placebo after 24 weeks of treatment; Parallel trend towards improvement in ADCS-ADL and ADCS-CGIC; Improvements in anxiety and hallucinations domains of the NPI-12 scale in a post hoc analysis
Alzheimer’s disease (Phase 3 studies) [76] NCT01955161, NCT02006641, and NCT02006654 Primary: Change from baseline in the ADAS-Cog
Other Key Endpoints: Change from baseline in the ADCS-ADL, ADCS-CGIC, MMSE and NPI-12
As augmentation therapy, no significant improvements in ADAS-Cog 11 scores as compared to placebo after 24 weeks of treatment; Similar observations were noted in other endpoints
Intepirdine Alzheimer’s disease
(Phase 2 study) [77]
NCT00224497 Primary: Change from baseline in the ADAS-Cog 11 scores and CIBIC+
Other Key Endpoints: Change from baseline in the MMSE and NPI-12
Significant improvement in the CIBIC+ and trend in the ADAS-Cog 11 scores was observed with 24 weeks of intepirdine treatment
Alzheimer’s disease
(Phase 2 studies) [78,79]
NCT00348192 and NCT00708552 Primary: Change from baseline in the ADAS-Cog 11 scores and CIBIC+
Other Key Endpoints: Change from baseline in the MMSE and ADCS-ADL
No significant effect was observed on the CIBIC+ or ADAS-Cog 11 scores at the end of 24 weeks of intepirdine treatment
Alzheimer’s disease
(Phase 2 study) [79]
NCT00710684 Primary: Change from baseline in the ADAS-Cog 11 scores and CDR-SB
Other Key Endpoints: Change from baseline in the MMSE and ADCS-ADL
As an add-on therapy to donepezil, beneficial effects of intepirdine were observed in the ADAS-Cog 11 scores at the end of 24 weeks and the effects were noted up to 48 weeks; No notable effects were observed on the CDR-SB
Alzheimer’s disease
(Phase 3 study) [80]
NCT02585934 Primary: Change from baseline in the ADAS-Cog 11 scores and ADCS-ADL
Other Key Endpoints: Change from baseline in the NPI-12
As an add-on therapy to donepezil, no beneficial effects of intepirdine were observed in the ADAS-Cog 11 or ADCS-ADL scores at the end of 24 weeks
Dementia with Lewy bodies
(Phase 2 study) [81]
NCT02669433 Primary: Change from baseline in the UPDRS–III total scores
Other Key Endpoints: Change from baseline in the ADAS-Cog 11 and CIBIC+
No beneficial effects of intepirdine were observed in the UPDRS–III total score at the end of 24 weeks
Landipirdine Parkinson’s disease dementia
(Phase 2 study) [82]
NCT02258152 Primary: Change from baseline in the CDRCOA total scores
Other Key Endpoints: Change from baseline in the ADCS-CGIC, MoCA and NPI-12
No beneficial effects of landipirdine were observed as an add-on treatment to cholinesterase inhibitor after 16 weeks of treatment;
Post hoc analysis suggested beneficial effects on apathy, anxiety, and irritability/lability
Latrepirdine Schizophrenia
(Phase 2 study)
[83]
Not available Key Endpoints: PANSS, CGI-S and NSA-16 As an add-on therapy, no beneficial effect of latrepirdine was observed in the PANSS total or sub scale scores; Latrepirdine showed statistically significant improvement in the NSA-16 scale
Alzheimer’s disease
(Pilot study) [44]
Not available Key Endpoint: Bukatina scale Treatment with latrepirdine was associated with improvements in cognitive function and reduction of NPS
Alzheimer’s disease
(Phase 2 study) [84]
NCT00377715 Primary: Change from baseline in the ADAS-Cog 11 scores
Other Key Endpoints: Change from baseline in the MMSE, ADCS-ADL and NPI-12
Significant improvement was observed on the ADAS-Cog 11, MMSE, ADCS-ADL and NPI-12 after 24 weeks of treatment
Alzheimer’s disease
(Phase 3 studies) [85]
NCT00675623 and NCT00829374 Primary: Change from baseline in the ADAS-Cog 11 scores and CIBIC+ (NCT00675623) or change from baseline in the ADAS-Cog 11 scores and ADCS-ADL (NCT00829374)
Other Key Endpoints: Change from baseline in the MMSE, ADCS-ADL and NPI-12
No significant effect of latrepirdine treatment was observed as standalone or add-on to donepezil after 26 or 52 weeks of treatment
Masupirdine Alzheimer’s disease
(Phase 2 study) [86,87,88]
NCT02580305 Primary: Change from baseline in the ADAS-Cog 11 total scores
Other Key Endpoints: Change from baseline in the ADCS-ADL, MMSE, CDR-SB and NPI-12
No beneficial effects of masupirdine were observed as an add-on treatment to donepezil and memantine in the ADAS-Cog 11 after 26 weeks of treatment; Post hoc analysis suggested potential impact of memantine on the efficacy, and potential beneficial effects on agitation/aggression and psychosis
Alzheimer’s disease Agitation
(Potentially pivotal study) [89]
NCT05397639 Primary: Change from baseline in the CMAI items scores aligning to the International Psychogeriatric Association agitation criteria domains
Other Key Endpoints: Change from baseline in the modified ADCS-CGI-C, MMSE and ADAS-Cog 11
Study in progress
SAM-760 Alzheimer’s disease
(Phase 2 study) [90]
NCT01712074 Primary: Change from baseline in the ADAS-Cog 13 total scores
Other Key Endpoints: Change from baseline in the COWAT, CFT and NPI-12
Trial was stopped after a futility analysis; No beneficial effect of SAM-760 treatment was observed after 12 weeks of treatment

ADAS-Cog—Alzheimer’s Disease Assessment Scale-Cognitive subscale; ADAS-Cog 11—Alzheimer’s Disease Assessment Scale-Cognitive subscale 11; ADAS-Cog 13—Alzheimer’s Disease Assessment Scale-Cognitive subscale 13; ADCS-ADL—Alzheimer’s Disease Cooperative Study—Activities of Daily Living; ADCS-CGIC—Alzheimer’s Disease Cooperative Study Clinical Global Impression of Change; BACS—Brief Assessment of Cognition in Schizophrenia; CANTAB—Cambridge Neuropsychological Test Automated Battery; CAT—Continuous Attention Task; CDRCOA—Computerized Drug Research Cognition Battery Continuity of Attention; CDR-SB—Clinical Dementia Rating scale Sum of Boxes; CFT—Category Fluency Test; CGI-I—Clinical Global Impression–Improvement; CGI-S—Clinical Global Impression–Severity; CIBIC+—Clinician’s Interview-Based Impression of Change with caregiver input; CMAI—Cohen-Mansfield Agitation Inventory; CogFu—Scale for Rating Functioning Related to Cognitive Impairment in Schizophrenia; COWAT—Controlled Oral Word Association Test; CPT—Continuous Performance Test; MMSE—Mini-Mental State Examination; MoCA—Montreal Cognitive Assessment. NPI-12—12-item Neuropsychiatric Inventory; NPS—Neuropsychiatric symptoms; NSA-16—16-item Negative Symptom Assessment; PANSS—Positive and Negative Syndrome Scale; PSPS—Personal and Social Performance Scale; UPDRS–III—Unified Parkinson’s Disease Rating Scale–Part III.