Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome (NCT NCT05307679)

NCT ID: NCT05307679

Last Updated: 2025-12-12

Results Overview

The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills for those with intellectual and developmental disabilities. Items consist of open-ended questions related to activities and behavior and are scored as 2 = Usually, 1 = Sometimes, and 0 = Never. Items requiring a binary response are scored as 2 = Yes, 0. Standard scores for the adaptive behavior composite range from 20-160; lower scores indicate lower adaptive functioning.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 7 participants
• Primary outcome timeframe: Baseline, Day 183, Day 365
• Results posted on: 2025-12-12

Participant Flow

Participant milestones

Measure	Placebo Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Overall Study STARTED	2	5
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	2	5

Reasons for withdrawal

Measure	Placebo Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Overall Study Study terminated by sponsor or physician decision	2	5

Baseline Characteristics

Participants were not consented on the collection of race and ethnicity data.

Baseline characteristics by cohort

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.	Total n=7 Participants Total of all reporting groups
Race (NIH/OMB) American Indian or Alaska Native	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Age, Continuous	8.0 Years STANDARD_DEVIATION 1.4 • n=2 Participants	8.8 Years STANDARD_DEVIATION 2.2 • n=5 Participants	8.6 Years STANDARD_DEVIATION 1.9 • n=7 Participants
Sex/Gender, Customized Female + Male	2 Count of Participants n=2 Participants	5 Count of Participants n=5 Participants	7 Count of Participants n=7 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Ethnicity (NIH/OMB) Not Hispanic or Latino	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Ethnicity (NIH/OMB) Unknown or Not Reported	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Race (NIH/OMB) Asian	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Race (NIH/OMB) Black or African American	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Race (NIH/OMB) White	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Race (NIH/OMB) More than one race	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.
Race (NIH/OMB) Unknown or Not Reported	—	—	0 Participants Participants were not consented on the collection of race and ethnicity data.

PRIMARY outcome

Timeframe: Baseline, Day 183, Day 365

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills for those with intellectual and developmental disabilities. Items consist of open-ended questions related to activities and behavior and are scored as 2 = Usually, 1 = Sometimes, and 0 = Never. Items requiring a binary response are scored as 2 = Yes, 0. Standard scores for the adaptive behavior composite range from 20-160; lower scores indicate lower adaptive functioning.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Vineland-3 Adaptive Behavior Scales, 3rd Edition Composite Scores Baseline	30.50 Units on a scale Interval 30.0 to 31.0	36.00 Units on a scale Interval 27.0 to 52.0
Vineland-3 Adaptive Behavior Scales, 3rd Edition Composite Scores Day 183	28.00 Units on a scale Interval 28.0 to 28.0	32.00 Units on a scale Interval 32.0 to 32.0
Vineland-3 Adaptive Behavior Scales, 3rd Edition Composite Scores Day 365	32.00 Units on a scale Interval 32.0 to 32.0	—

SECONDARY outcome

Timeframe: Baseline, Day 183, Day 365

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills for those with intellectual and developmental disabilities. Items consist of open-ended questions related to activities and behavior and are scored as 2 = Usually, 1 = Sometimes, and 0 = Never. Items requiring a binary response are scored as 2 = Yes, 0. V-scale scores for the gross and fine motor domains range from 1-24; lower scores indicate lower adaptive functioning.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=2 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Vineland-3 Gross and Fine Motor Subdomains Scores Gross Motor V-Scale Score - Day 365	1.00 Units on a scale Interval 1.0 to 1.0	—
Vineland-3 Gross and Fine Motor Subdomains Scores Gross Motor V-Scale Score - Baseline	1.00 Units on a scale Interval 1.0 to 1.0	4.50 Units on a scale Interval 1.0 to 8.0
Vineland-3 Gross and Fine Motor Subdomains Scores Gross Motor V-Scale Score - Day 183	4.00 Units on a scale Interval 4.0 to 4.0	—
Vineland-3 Gross and Fine Motor Subdomains Scores Fine Motor V-Scale Score - Baseline	1.00 Units on a scale Interval 1.0 to 1.0	3.00 Units on a scale Interval 1.0 to 5.0
Vineland-3 Gross and Fine Motor Subdomains Scores Fine Motor V-Scale Score - Day 183	1.00 Units on a scale Interval 1.0 to 1.0	—
Vineland-3 Gross and Fine Motor Subdomains Scores Fine Motor V-Scale Score - Day 365	1.00 Units on a scale Interval 1.0 to 1.0	—

SECONDARY outcome

Timeframe: Baseline, Day 183, Day 365

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills for those with intellectual and developmental disabilities. Items consist of open-ended questions related to activities and behavior and are scored as 2 = Usually, 1 = Sometimes, and 0 = Never. Items requiring a binary response are scored as 2 = Yes, 0. V-scale scores for the expressive and receptive communication subdomains range from 1-24; lower scores indicate lower adaptive functioning.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Vineland 3 Expressive and Receptive Communication Subdomains Expressive Communication V-Scale Score - Day 365	1.00 Units on a scale Interval 1.0 to 1.0	—
Vineland 3 Expressive and Receptive Communication Subdomains Receptive Communication V-Scale Score - Day 365	1.00 Units on a scale Interval 1.0 to 1.0	—
Vineland 3 Expressive and Receptive Communication Subdomains Expressive Communication V-Scale Score - Baseline	1.00 Units on a scale Interval 1.0 to 1.0	1.00 Units on a scale Interval 1.0 to 11.0
Vineland 3 Expressive and Receptive Communication Subdomains Expressive Communication V-Scale Score - Day 183	1.00 Units on a scale Interval 1.0 to 1.0	1.00 Units on a scale Interval 1.0 to 1.0
Vineland 3 Expressive and Receptive Communication Subdomains Receptive Communication V-Scale Score - Baseline	1.00 Units on a scale Interval 1.0 to 1.0	1.00 Units on a scale Interval 1.0 to 6.0
Vineland 3 Expressive and Receptive Communication Subdomains Receptive Communication V-Scale Score - Day 183	1.00 Units on a scale Interval 1.0 to 1.0	1.00 Units on a scale Interval 1.0 to 1.0

SECONDARY outcome

Timeframe: Baseline, Day 153, Day 365

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills for those with intellectual and developmental disabilities. Items consist of open-ended questions related to activities and behavior and are scored as 2 = Usually, 1 = Sometimes, and 0 = Never. Items requiring a binary response are scored as 2 = Yes, 0. V-scale scores for the play and leisure time, and interpersonal relationships subdomains range from 1-24; lower scores indicate lower adaptive functioning.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Vineland-3 Play and Leisure Time and Interpersonal Relationships Subdomains Play and Leisure V-Scale Score - Baseline	1.00 Units on a scale Interval 1.0 to 1.0	1.00 Units on a scale Interval 1.0 to 9.0
Vineland-3 Play and Leisure Time and Interpersonal Relationships Subdomains Play and Leisure V-Scale Score - Day 183	1.00 Units on a scale Interval 1.0 to 1.0	1.00 Units on a scale Interval 1.0 to 1.0
Vineland-3 Play and Leisure Time and Interpersonal Relationships Subdomains Play and Leisure V-Scale Score - Day 365	1.00 Units on a scale Interval 1.0 to 1.0	—
Vineland-3 Play and Leisure Time and Interpersonal Relationships Subdomains Interpersonal Relationships V-Scale Score - Baseline	1.50 Units on a scale Interval 1.0 to 2.0	1.00 Units on a scale Interval 1.0 to 9.0
Vineland-3 Play and Leisure Time and Interpersonal Relationships Subdomains Interpersonal Relationships V-Scale Score - Day 183	3.00 Units on a scale Interval 3.0 to 3.0	1.00 Units on a scale Interval 1.0 to 1.0
Vineland-3 Play and Leisure Time and Interpersonal Relationships Subdomains Interpersonal Relationships V-Scale Score - Day 365	3.00 Units on a scale Interval 3.0 to 3.0	—

SECONDARY outcome

Timeframe: Baseline, Day 183, Day 365

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The MSEL are designed for a certified rater to provide an assessment of cognitive ability and motor development of typically developing children from birth through age 68 months. It was administered to all participants in this study irrespective of their chronological age. The instrument consists of 124 items across five scales measuring Gross Motor, Visual Reception, Fine Motor, Expressive Language, and Receptive Language. Each item is scored from 0-5 points with total raw score ranges of 0-100 (gross motor) and 0-78 (fine motor). Lower score indicate lower developmental abilities; higher scores indicate greater developmental abilities.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Mullen Scales of Early Learning (MSEL) Gross and Fine Motor Domains Gross Motor - Baseline	20.50 Units on a scale Interval 14.0 to 27.0	19.00 Units on a scale Interval 13.0 to 35.0
Mullen Scales of Early Learning (MSEL) Gross and Fine Motor Domains Gross Motor - Day 183	24.00 Units on a scale Interval 24.0 to 24.0	18.00 Units on a scale Interval 18.0 to 18.0
Mullen Scales of Early Learning (MSEL) Gross and Fine Motor Domains Gross Motor - Day 365	1.00 Units on a scale Interval 1.0 to 1.0	—
Mullen Scales of Early Learning (MSEL) Gross and Fine Motor Domains Fine Motor - Baseline	15.50 Units on a scale Interval 13.0 to 18.0	15.00 Units on a scale Interval 6.0 to 30.0
Mullen Scales of Early Learning (MSEL) Gross and Fine Motor Domains Fine Motor - Day 183	18.00 Units on a scale Interval 18.0 to 18.0	9.00 Units on a scale Interval 9.0 to 9.0
Mullen Scales of Early Learning (MSEL) Gross and Fine Motor Domains Fine Motor - Day 365	11.00 Units on a scale Interval 11.0 to 11.0	—

SECONDARY outcome

Timeframe: Baseline, Day 183

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The MSEL are designed for a certified rater to provide an assessment of cognitive ability and motor development of typically developing children from birth through age 68 months. It was administered to all participants in this study irrespective of their chronological age. The instrument consists of 124 items across five scales measuring Gross Motor, Visual Reception, Fine Motor, Expressive Language, and Receptive Language. Each item is scored from 0-5 points with a total raw score range of 0-50. Lower score indicate lower developmental abilities; higher scores indicate greater developmental abilities.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
MSEL Visual Reception Domain Scores Baseline	14.00 Units on a scale Interval 11.0 to 17.0	4.00 Units on a scale Interval 2.0 to 42.0
MSEL Visual Reception Domain Scores Day 183	21.00 Units on a scale Interval 21.0 to 21.0	5.00 Units on a scale Interval 5.0 to 5.0
MSEL Visual Reception Domain Scores Day 365	13.00 Units on a scale Interval 13.0 to 13.0	—

SECONDARY outcome

Timeframe: Baseline, Day 183

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The MSEL are designed for a certified rater to provide an assessment of cognitive ability and motor development of typically developing children from birth through age 68 months. It was administered to all participants in this study irrespective of their chronological age. The instrument consists of 124 items across five scales measuring Gross Motor, Visual Reception, Fine Motor, Expressive Language, and Receptive Language. Each item is scored from 0-5 points with a total raw score range of 0-50 (expressive language) or 0-48 (receptive language). Lower score indicate lower developmental abilities; higher scores indicate greater developmental abilities.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
MSEL Expressive and Receptive Language Subdomains Expressive Language - Baseline	12.50 Units on a scale Interval 12.0 to 13.0	17.00 Units on a scale Interval 6.0 to 29.0
MSEL Expressive and Receptive Language Subdomains Expressive Language - Day 183	23.00 Units on a scale Interval 23.0 to 23.0	6.00 Units on a scale Interval 6.0 to 6.0
MSEL Expressive and Receptive Language Subdomains Expressive Language - Day 365	7.00 Units on a scale Interval 7.0 to 7.0	—
MSEL Expressive and Receptive Language Subdomains Receptive Language - Baseline	10.00 Units on a scale Interval 7.0 to 13.0	16.00 Units on a scale Interval 9.0 to 28.0
MSEL Expressive and Receptive Language Subdomains Receptive Language - Day 183	17.00 Units on a scale Interval 17.0 to 17.0	11.00 Units on a scale Interval 11.0 to 11.0
MSEL Expressive and Receptive Language Subdomains Receptive Language - Day 365	13.00 Units on a scale Interval 13.0 to 13.0	—

SECONDARY outcome

Timeframe: Baseline until Day 395, or early termination (prior to Day 395)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The Dup15q CGI-S is a 10-domain clinician-rated measure on a 6-point scale that measures global severity of illness at a given point in time. The ten domains are seizures, expressive communication difficulties, fine motor skills difficulties, gross motor skills difficulties, cognitive/intellectual impairment, impairment in activities of daily living/self-care, socialization, maladaptive behavior, sleep difficulties, and overall severity. Response options are 1-none, 2-very mild, 3-mild, 4-moderate, 5-severe, and 6-very severe.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Day 28	4.00 Units on a scale Interval 4.0 to 4.0	4.00 Units on a scale Interval 3.0 to 6.0
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Baseline	4.00 Units on a scale Interval 4.0 to 4.0	4.00 Units on a scale Interval 4.0 to 6.0
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Day 92	4.00 Units on a scale Interval 4.0 to 4.0	4.00 Units on a scale Interval 4.0 to 6.0
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Day 183	4.00 Units on a scale Interval 4.0 to 4.0	4.00 Units on a scale Interval 4.0 to 4.0
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Day 274	4.00 Units on a scale Interval 4.0 to 4.0	—
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Day 365	4.00 Units on a scale Interval 4.0 to 4.0	—
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Follow-Up Day 395	—	5.00 Units on a scale Interval 4.0 to 6.0
Dup15q Syndrome Clinician Global Impression of Severity (CGI-S) Scale Scores Early Termination	—	6.00 Units on a scale Interval 6.0 to 6.0

SECONDARY outcome

Timeframe: Day 28 - Day 395 or early termination (prior to Day 395)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The Dup15q CGI-C is a 10-domain clinician-rated measure on a 7-point scale that assesses the clinician's impression of change in illness compared with baseline. The ten domains are seizures, expressive communication difficulties, fine motor skills difficulties, gross motor skills difficulties, cognitive/intellectual impairment, impairment in activities of daily living/self-care, socialization, maladaptive behavior, sleep difficulties, and overall severity. Response options are 1-very much improved, 2-much improved, 3-minimally improved, 4-no change, 5-minimally worse, 6-much worse, and 7-very much worse. The CGI does not yield a global score.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Day 28	3.50 Units on a scale Interval 3.0 to 4.0	3.00 Units on a scale Interval 2.0 to 3.0
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Day 92	3.00 Units on a scale Interval 3.0 to 3.0	3.00 Units on a scale Interval 2.0 to 4.0
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Day 183	4.00 Units on a scale Interval 4.0 to 4.0	3.00 Units on a scale Interval 3.0 to 3.0
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Day 274	4.00 Units on a scale Interval 4.0 to 4.0	—
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Day 365	3.00 Units on a scale Interval 3.0 to 3.0	—
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Follow-Up Day 395	—	3.00 Units on a scale Interval 3.0 to 4.0
Dup15q Syndrome Clinician Global Impression of Change Scale (CGI-C) Scores Early Termination	—	4.00 Units on a scale Interval 4.0 to 4.0

SECONDARY outcome

Timeframe: Baseline - Day 365, or early termination (prior to Day 365)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). The irritability domain score range is 0-45, with higher score indicating greater severity.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Baseline	11.00 Units on a scale Interval 11.0 to 11.0	15.00 Units on a scale Interval 3.0 to 40.0
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Day 28	10.00 Units on a scale Interval 6.0 to 14.0	15.00 Units on a scale Interval 5.0 to 18.0
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Day 92	17.00 Units on a scale Interval 17.0 to 17.0	16.00 Units on a scale Interval 5.0 to 23.0
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Day 183	20.00 Units on a scale Interval 20.0 to 20.0	28.00 Units on a scale Interval 28.0 to 28.0
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Day 274	16.00 Units on a scale Interval 16.0 to 16.0	—
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Day 365	13.00 Units on a scale Interval 13.0 to 13.0	—
Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) Domain Scores - Irritability Early Termination	—	3.00 Units on a scale Interval 3.0 to 3.0

SECONDARY outcome

Timeframe: Baseline - Day 365, or early termination (prior to Day 365)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). The social withdrawal domain score range is 0-48, with higher scores indicating greater severity.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
ABC-2-C Domain Scores - Social Withdrawal Baseline	14.50 Units on a scale Interval 11.0 to 18.0	16.00 Units on a scale Interval 2.0 to 25.0
ABC-2-C Domain Scores - Social Withdrawal Day 28	7.50 Units on a scale Interval 6.0 to 9.0	14.00 Units on a scale Interval 7.0 to 17.0
ABC-2-C Domain Scores - Social Withdrawal Day 92	10.00 Units on a scale Interval 10.0 to 10.0	12.00 Units on a scale Interval 6.0 to 24.0
ABC-2-C Domain Scores - Social Withdrawal Day 183	10.00 Units on a scale Interval 10.0 to 10.0	7.00 Units on a scale Interval 7.0 to 7.0
ABC-2-C Domain Scores - Social Withdrawal Day 274	2.00 Units on a scale Interval 2.0 to 2.0	—
ABC-2-C Domain Scores - Social Withdrawal Day 365	4.00 Units on a scale Interval 4.0 to 4.0	—
ABC-2-C Domain Scores - Social Withdrawal Early Termination	—	22.00 Units on a scale Interval 22.0 to 22.0

SECONDARY outcome

Timeframe: Baseline - Day 365, or early termination (prior to Day 365)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). The stereotypic behavior subdomain score range is 0-21, with higher scores indicating greater severity.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
ABC-2-C Domain Scores - Stereotypic Behavior Baseline	8.00 Units on a scale Interval 8.0 to 8.0	6.00 Units on a scale Interval 0.0 to 15.0
ABC-2-C Domain Scores - Stereotypic Behavior Day 28	5.50 Units on a scale Interval 5.0 to 6.0	13.00 Units on a scale Interval 7.0 to 16.0
ABC-2-C Domain Scores - Stereotypic Behavior Day 92	6.00 Units on a scale Interval 6.0 to 6.0	14.00 Units on a scale Interval 8.0 to 15.0
ABC-2-C Domain Scores - Stereotypic Behavior Day 183	4.00 Units on a scale Interval 4.0 to 4.0	6.00 Units on a scale Interval 6.0 to 6.0
ABC-2-C Domain Scores - Stereotypic Behavior Day 274	8.00 Units on a scale Interval 8.0 to 8.0	—
ABC-2-C Domain Scores - Stereotypic Behavior Day 365	2.00 Units on a scale Interval 2.0 to 2.0	—
ABC-2-C Domain Scores - Stereotypic Behavior Early Termination	—	11.00 Units on a scale Interval 11.0 to 11.0

SECONDARY outcome

Timeframe: Baseline until Day 365, or early termination (prior to Day 365)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). The hyperactivity/non-compliance subdomain score range is 0-45, with higher scores indicating greater severity.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Baseline	32.00 Units on a scale Interval 23.0 to 41.0	19.00 Units on a scale Interval 13.0 to 38.0
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Day 28	12.00 Units on a scale Interval 6.0 to 18.0	22.00 Units on a scale Interval 15.0 to 33.0
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Day 92	40.00 Units on a scale Interval 40.0 to 40.0	18.00 Units on a scale Interval 14.0 to 35.0
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Day 183	39.00 Units on a scale Interval 39.0 to 39.0	18.00 Units on a scale Interval 18.0 to 18.0
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Day 274	40.00 Units on a scale Interval 40.0 to 40.0	—
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Day 365	33.00 Units on a scale Interval 33.0 to 33.0	—
ABC-2-C Domain Scores - Hyperactivity/Non-Compliance Early Termination	—	14.00 Units on a scale Interval 14.0 to 14.0

SECONDARY outcome

Timeframe: Baseline until Day 365, or early termination (prior to Day 365)

Population: ITT population: All randomized participants who received ≥1 dose. Participants were allocated to the treatment arm they were randomized to.

The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). The inappropriate speech subdomain score range is 0-12, with higher scores indicating greater severity.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
ABC-2-C Domain Scores - Inappropriate Speech Baseline	5.00 Units on a scale Interval 2.0 to 8.0	2.00 Units on a scale Interval 0.0 to 10.0
ABC-2-C Domain Scores - Inappropriate Speech Day 28	1.50 Units on a scale Interval 0.0 to 3.0	0.00 Units on a scale Interval 0.0 to 2.0
ABC-2-C Domain Scores - Inappropriate Speech Day 92	3.00 Units on a scale Interval 3.0 to 3.0	0.00 Units on a scale Interval 0.0 to 8.0
ABC-2-C Domain Scores - Inappropriate Speech Day 183	1.00 Units on a scale Interval 1.0 to 1.0	0.00 Units on a scale Interval 0.0 to 0.0
ABC-2-C Domain Scores - Inappropriate Speech Day 274	3.00 Units on a scale Interval 3.0 to 3.0	—
ABC-2-C Domain Scores - Inappropriate Speech Day 365	2.00 Units on a scale Interval 2.0 to 2.0	—
ABC-2-C Domain Scores - Inappropriate Speech Early Termination	—	0.00 Units on a scale Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Up to 52 weeks

Population: The safety analysis population is the same as the ITT population, assuming treatment is received as assigned at randomization.

An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) AEs	2 Count of Participants	5 Count of Participants
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) SAEs	0 Count of Participants	1 Count of Participants

SECONDARY outcome

Timeframe: Day 1 - Day 183

Population: The pharmacokinetic (PK) population consisted of all participants who received at least one dose of study treatment and who had data from at least one postdose sample. Only assessment time points with \> 1 participant are shown due to concerns that certain demographic characteristics that may be present in only one treatment arm, or the duration a participant was in the trial, present an unacceptable risk of participant re-identification.

Outcome measures

Measure	Placebo n=5 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Plasma Concentration of Basmisanil Day 1 - Hour 1.5	2520 ng/mL Standard Deviation 965	—
Plasma Concentration of Basmisanil Day 1 - Hour 3.5	2220 ng/mL Standard Deviation 955	—
Plasma Concentration of Basmisanil Day 1 - Hour 5.5	1640 ng/mL Standard Deviation 756	—
Plasma Concentration of Basmisanil Day 1 - Hour 7.5	1040 ng/mL Standard Deviation 392	—
Plasma Concentration of Basmisanil Day 1 - Hour 9	791 ng/mL Standard Deviation 368	—
Plasma Concentration of Basmisanil Day 2 - Hour 0	731 ng/mL Standard Deviation 535	—
Plasma Concentration of Basmisanil Day 2 - Hour 1.5	2380 ng/mL Standard Deviation 576	—
Plasma Concentration of Basmisanil Day 2 - Hour 3.5	2210 ng/mL Standard Deviation 864	—
Plasma Concentration of Basmisanil Day 14 - Hour -1.5	1210 ng/mL Standard Deviation 1230	—
Plasma Concentration of Basmisanil Day 14 - Hour 0	902 ng/mL Standard Deviation 973	—
Plasma Concentration of Basmisanil Day 14 - Hour 1.5	2480 ng/mL Standard Deviation 355	—
Plasma Concentration of Basmisanil Day 14 - Hour 3.5	2160 ng/mL Standard Deviation 698	—
Plasma Concentration of Basmisanil Day 92 - Hour 0	896 ng/mL Standard Deviation 689	—

SECONDARY outcome

Timeframe: Day 1 - Day 183

Population: The pharmacokinetic (PK) population consisted of all participants who received at least one dose of study treatment and who had data from at least one postdose sample. Only assessment time points with \> 1 participant are shown due to concerns that certain demographic characteristics that may be present in only one treatment arm, or the duration a participant was in the trial, present an unacceptable risk of participant re-identification.

Outcome measures

Measure	Placebo n=5 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Plasma Concentration of the Basmisanil Metabolite M1 Day 14 - 1.5	1510 ng/mL Standard Deviation 413	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 14 - Hour 3.5	1250 ng/mL Standard Deviation 540	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 92 - Hour 0	648 ng/mL Standard Deviation 522	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 1 - Hour 1.5	1410 ng/mL Standard Deviation 585	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 1 - Hour 3.5	1510 ng/mL Standard Deviation 417	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 1 - Hour 5.5	985 ng/mL Standard Deviation 335	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 1 - Hour 7.5	675 ng/mL Standard Deviation 221	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 1 - Hour 9	535 ng/mL Standard Deviation 173	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 2 - Hour 0	664 ng/mL Standard Deviation 568	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 2 - Hour 1.5	1330 ng/mL Standard Deviation 358	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 2 - Hour 3.5	1410 ng/mL Standard Deviation 506	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 14 - Hour -1.5	1400 ng/mL Standard Deviation 1410	—
Plasma Concentration of the Basmisanil Metabolite M1 Day 14 - Hour 0	1060 ng/mL Standard Deviation 1470	—

SECONDARY outcome

Timeframe: Baseline, Day 14

This is the average of the EEG parameter 10 x Log_10 of the total beta power between 16 and 32 Hz in microVolt^2.

Outcome measures

Measure	Placebo n=2 Participants Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 Participants Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Quantitative EEG (qEEG) Beta-band Power Baseline	9.47 µV2 Standard Deviation 0.21	11.39 µV2 Standard Deviation 1.55
Quantitative EEG (qEEG) Beta-band Power Day 14	9.44 µV2 Standard Deviation 0.37	9.74 µV2 Standard Deviation 1.05

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Basmisanil

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=2 participants at risk Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 participants at risk Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
General disorders Unspecified SAE	0.00% 0/2 • Baseline - Day 365 Observations where n \</= 1 were combined into "unspecified" categories due to an unacceptable risk of participant re-identification.	20.0% 1/5 • Number of events 3 • Baseline - Day 365 Observations where n \</= 1 were combined into "unspecified" categories due to an unacceptable risk of participant re-identification.

Other adverse events

Measure	Placebo n=2 participants at risk Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.	Basmisanil n=5 participants at risk Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
Injury, poisoning and procedural complications Incorrect dose administered	100.0% 2/2 • Number of events 6 • Baseline - Day 365 Observations where n \</= 1 were combined into "unspecified" categories due to an unacceptable risk of participant re-identification.	60.0% 3/5 • Number of events 7 • Baseline - Day 365 Observations where n \</= 1 were combined into "unspecified" categories due to an unacceptable risk of participant re-identification.
General disorders Unspecified AE	50.0% 1/2 • Number of events 2 • Baseline - Day 365 Observations where n \</= 1 were combined into "unspecified" categories due to an unacceptable risk of participant re-identification.	40.0% 2/5 • Number of events 7 • Baseline - Day 365 Observations where n \</= 1 were combined into "unspecified" categories due to an unacceptable risk of participant re-identification.

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 1-800-821-8590

Email: genentech@druginfo.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
• Publication restrictions are in place

Restriction type: OTHER