An Open-Label Phase 2a Study to Evaluate the Safety, Tolerability, and Efficacy of Vafidemstat in Adults with Phelan-McDermid Syndrome (PMS) (HOPE-2).

Condition studied

Phelan McDermid Syndrome

Eligibility

Primary endpoint

Number, frequency and severity of Treatment Emergent Adverse Events (TEAEs), including Adverse Events of Special Interest (AESIs)., Number, frequency and severity of serious TEAEs, including AESIs., Number and percentage of withdrawn Participants due to TEAEs, including AESIs., Frequency of clinically relevant abnormalities in physical examination findings, vital signs and ECG parameters., Frequency of clinical laboratory parameters (haematology and chemistry) of potential clinical concern., Number and percentage of Participants exhibiting suicidal ideation or behaviours, as evaluated by the Columbia - Suicide Severity Rating Scale (C-SSRS)., Concomitant medication use.

Endpoint detail

Change from Baseline (Visit 2) in the: - Aberrant Behaviour Checklist (ABC) Irritability Subscale. - Clinical Global Impression of Severity - Anger and Aggression (CGI-S A/A)., Change from Baseline (Visit 2) in the: - Repetitive Behaviour Scale-Revised (RBS-R). - Phelan-McDermid Syndrome Assessment of Severity (PMSA-S). - ABC Subscales: o Stereotypic Behaviour. o Hyperactivity/Non-compliance. o Inappropriate Speech. o Social Withdrawal.