Newleos Therapeutics, Inc.
The primary goal of this Phase 2 clinical trial is to determine the effects of a selective negative allosteric modulator (NAM) of GABAA α5 receptor, Basmisanil (NTX-1511), on brain wave (EEG) characteristics in children and adolescents with Dup15q syndrome. The main questions this trial aims to answer are: * How does NTX-1511 affect brain waves (as measured by EEG) in subjects with Dup15q syndrome? * Does NTX-1511 impact other developmental features of subjects with Dup15q syndrome? Researchers will compare the effects of NTX-1511 with matching placebo (look-alike granules that contain no drug). Participants will: * Take NTX-1511 or matching placebo 3 times every day for 12 weeks * Visit the clinic 7 times over the course of 23 weeks
Key Inclusion Criteria: * Parent, caregiver, or legally authorized representatives has provided written informed consent for the study and is willing to comply with all requirements of the protocol * Male or female aged 2 to 14 years inclusive at time of informed consent signature * Documented duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2.-q13.1 (BP2-BP3) segment. * Dup15q syndrome Clinician Global Impression of Severity Scale (Dup15q CGI-S) overall severity score ≥ 3 (at least mildly impaired) Key Exclusion Criteria: * Pathogenic or likely pathogenic genomic v…
Three times daily (TID) x 12 weeks
Three times daily (TID) x 12 weeks
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