Study of the Drug BTD-001 in Young Adults and Adolescents with Down Syndrome

INTERVENTION: Participants (13 to 35 yrs in age) will be enrolled. Following screening, a visit prior to first dose will establish baseline measurements of cognitive function, adaptive behaviour scoring and evoked response measurements. Eligible participants will be randomised to one of the following treatments: Treatment A ‐ 50mg BTD‐001 taken orally twice daily, as oral solution. Treatment B ‐ 100mg BTD‐001 taken orally twice daily, as oral solution. Treatment C ‐ Placebo taken orally twice daily, as oral solution All study treatments will be made up as a flavoured drink solution. Study drug will be given for 12 weeks. CONDITION: Down syndrome PRIMARY OUTCOME: Safety and tolerability of BTD‐001 twice daily oral as assessed by: physical and neurological examinations; laboratory safety tests; ECG; vital signs; adverse event collection. Adverse events are any untoward medical occurrences in study participants administered study treatment. ; Based on animal studies and the experience of other people who have received the active ingredient in BTD‐001, side effects that may occur include feelings of anxiety and difficulty with sleeping. At very high doses, seizures (fits) and gastrointestinal effects (nausea and vomiting) have been reported to occur. Please note that these side effects have occurred at doses 10‐20 times greater than those being tested in this study. SECONDARY OUTCOME: Cognitive, and behavioural/functional effects of BTD‐001 in persons with Down syndrome as assessed by psychometric and functional measures. Psychometric measures include standardise tests assessments for functional and adaptative behavior and language. These include: Vineland adaptive behavior scale (VABS‐II), Behavior rating inventory of executive function (BRIEF), and Clinical Evaluation of Language Functions (CELF4). Cognitive test assessments will be performed using a computer based testing system [CANTAB(Registered Trademark)]. Effects of BTD‐001 on auditory event related potentials (ERP) in persons with Down syndrome. Auditory ERP will be assessed by electroencephalography (EEG). INCLUSION CRITERIA: 1. Males and females, age 13–35 years with Down syndrome 2. Body weight greater than 40 kg AND BMI less than 40.0 kg/m2 3. Ability to cooperate and complete required tests and procedures. 4. Capable of undergoing the cognitive test battery and evoked response potential testing. 5. Negative pregnancy test AND non‐lactating (females only) 6. If sexually active and female, the person with DS must be surgically sterile OR using reliable contraception 7. Laboratory findings, vital signs and ECG within normal limits or any abnormalities judged clinically not significant 8. Stable medical condition For Parents/Guardians 1. Parents or guardians of the person with DS must understand the study, give written and dated informed consent/assent, and successfully complete behavioural assessment interviews. 2. Parent or guardian agree to accompany the subject to all visits, be capable of supervising and reporting study drug use and adequately reporting the subject’s functional stat