This is a Study to Investigate the Effects and Safety of Codabakalner for the Treatment of Fragile X Syndrome in Male Participants and Adult Participants with Autism Spectrum Disorders
Phase 2b: Adult males aged 18 to 45 years, inclusive Phase 3: Males aged 13-45 years, inclusive Mental age >/= 36mon
Diagnosis of Fragile X as confirmed with genetic testing and Stanford Binet testing or
Confirmed diagnosis ASD per DSM5-TR and EEG criteria
Patient must have caregiver
Any physical or psychological condition that prohibits study completion
Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months.
Auditory or visual impairments that cannot be corrected
History of suicidal behaviour or suicidal ideation
Parallel
Codabakalner
Drugsynthetic small molecule
Placebo
DrugPlacebo
Phase 2b: FXS- Change from baseline in Total Cognition Composite Change Sensitive Score from the NIH-TCB
The NIH Toolbox (NIH-TCB) Total Cognition Composite Change-Sensitive Score (CSS) is used to measure cognitive change over time. Higher scores indicate higher level of functioning. The scores typically span across the developmental range, often extending well above and below the 500-point mark depending on the age and cognitive ability of the individual.
Phase 2b: ASD - Change from Baseline at Day 28 on the Vineland Adaptive Behavior Scales in ASD(Vineland-III) Adaptive Behavior Composite Score
Vineland Adaptive Behavior Composite (ABC) have a mean of 100 and a standard deviation of 15, where scores from 85 to 115 reflect expected or average adaptive functioning
Phase 3: FXS - Change from baseline in Total Cognition Composite score from the NIH-TCB
The NIH Toolbox (NIH-TCB) Total Cognition Composite Change-Sensitive Score (CSS) is used to measure cognitive change over time. It is calculated by combining the Crystallized Composite (reading/vocabulary) and Fluid Composite (memory/executive function/speed) scores. Higher scores indicate higher level of functioning.
Phase 2b: FXS- Change from baseline in Fluid Reasoning Composite Change Sensitive Scores from the NIH-TCB
The Fluid Cognition Composite (FCC) from the NIH Toolbox Cognition Battery (NIHTB-CB) is designed to measure changes to neurological functioning. It combines scores from five tests (Dimensional Change Card Sort, Flanker, List Sorting, Pattern Comparison, and Picture Sequence Memory) to provide a measure of executive function, memory, and processing speed. Higher scores indicate higher level of functioning. Superior ability is 130, average is approx. 100 and less than 70 is impairment.
Phase 2b: FXS- Change from baseline in Flanker Scores from the NIH-TCB
The Flanker test measures executive attention, inhibitory control, and attention by having participants identify the direction of a central arrow (or fish for young children) while ignoring surrounding flanking stimuli. Yields a standardized score scaled with a mean of 100 and a standard deviation of 15 (up to a maximum score of 130)
Phase 2b- FXS Change from Baseline at Day 28 on the Vineland Adaptive Behavior Scales (Vineland-III) Adaptive Behavior Composite Score
Vineland Adaptive Behavior Composite (ABC) have a mean of 100 and a standard deviation of 15, where scores from 85 to 115 reflect expected or average adaptive functioning
Phase 2b: FXS and ASD: Change from baseline at Day 28 in Clinical global impression of severity (CGI-S) as assessed by the investigator
This is a 7-point clinician-rated tool used to measure how ill a patient is at the time of assessment
This trial is not recruiting at the moment. You can still explore other options:
