A Phase II Study of Basmisanil in Children and Adolescents With Dup15q Syndrome
ConditionDup15Q Syndrome
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age2-14
SponsorNewleos Therapeutics, Inc.
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
Pathogenic or likely pathogenic genomic variant outside of the BP2-BP3 segment. Individuals with an isodicentric 15q chromosome and a history of epilepsy
Uncontrolled epilepsy (in individuals with interstitial BP2-BP3 duplication or triplication) indicated by: - Use of rescue medication(s) to treat more than one seizure episode or seizure cluster per month, on average in the past 6 months, or -Concomitant chronic use of more than four anti-epileptic medications, or -Status epilepticus within the past 6 months requiring hospitalization for treatment of the status epilepticus, or -Any implanted devices to treat drug-resistant epilepsy
Changes to medications within 6-8 weeks prior to screening
Changes to non-pharmacological/behavioral therapies within 6 weeks prior to screening
Trial design
Design model
Parallel
Treatments tested in this trial
Basmisanil
DrugThree times daily (TID) x 12 weeks
Placebo
DrugThree times daily (TID) x 12 weeks
Treatment groups
30 Participants
are divided into 2 treatment groupsGroup A: Experimental: NTX-1511Active comparator 1 intervention
Group B: Placebo Comparator: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Change in quantitative electroencephalogram (qEEG) beta-band power
Time frame
Baseline to Week 12Secondary outcomes
1
Change in Dup15q Clinical global impression of severity (CGI-s) and change (CGI-C)
The Dup15q Syndrome Clinician Global Impression of Severity (Dup15q CGI-S) is a clinician-rated measure of global severity of illness at a given point in time. Each domain scoreranges from 1-6. Higher scores indicate higher severity of illness. The Dup15q Syndrome Clinician Global Impression of Change (Dup15q CGI-C) is a clinician-rated measure assessing the clinician's impression of change in illness compared with baseline. Each domain score ranges from 1-7. Lower scores indicate improved symptoms, and higher scores indicate worsening symptoms.
Time frame
Baseline to Week 122
Change in Vineland Adaptive Behavior Scales -Third Edition (Vineland-3) subdomain Growth Scale Value (GSV) scores
The Vineland Adaptive Behavior Scales -Third Edition (Vineland-3) measures adaptive behavior in 3 domains: Communication (subdomains Expressive, Reception, and Written), Socialization (subdomains Interpersonal, Play \& Leisure, Coping Skills), and Daily Living Skills (Personal, Domestic, Community), and also yields an overall Adaptive Behavior Composite score. The domains and the Composite are reported as a standard score (population mean 100; standard deviation 15; range 20-140). The subdomains are reported as v-scale scores (population mean 10, standard deviation 3; range 1-24). Higher scores indicate higher adaptive behavior abilities
Time frame
Baseline to Week 123
Change in Behavior Assessment System for Children - Third Edition (BASC-3) composite and scale scores
The Behavior Assessment System for Children (BASC-3) assesses behavioral and emotional concerns. It has multiple scales: Externalizing Problems (subscales Hyperactivity, Aggression; Conduct Problems); Internalizing Problems (Anxiety, Depression Somatization); additional subscales of Attention Problems, Atypicality, and Withdrawal, and an overall Behavioral Symptoms Index. Each of these scales and subscales is scored as a T-score (population mean of 50, standard deviation of 10; range 20-120). Higher T-scores indicate greater behavioral challenges.
Time frame
Baseline to Week 124
Change in Aberrant Behavior Checklist - Second Edition Community Version (ABC-2-C) domain scores
The Aberrant Behavior Checklist (ABC-2-C) domain scores assess challenging behaviors. The domain scores each have different ranges: Irritability from 0-45; Lethargy/Social Withdrawal 0-48; Stereotypy 0-21; Hyperactivity 0-48; Inappropriate Speech 0-12. Higher scores indicate more severe symptoms.
Time frame
Baseline to Week 12Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: