Study of the Drug BTD-001 in Young Adults and Adolescents with Down Syndrome
- Conditions
- Down syndromeHuman Genetics and Inherited Disorders - Down's syndrome
- Registration Number
- ACTRN12612000652875
- Lead Sponsor
- ovotech (Australia) Pty Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 90
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 status.
1. Current use of anti-epileptics, focal seizure disorder, syncope, unexplained loss of consciousness at any time OR seizure in the past 3 years.
2. History of or concurrent neurological disorders interfering with cognition.
3. History or diagnosis of clinically significant psychiatric or neurological disease.
4. Neuroleptic agent, bilobalide-containing product OR benzodiazepine use in last 4 weeks
5. Significant endocrine abnormality
6. Subjects with hypothyroidism are excluded unless they have received a stable dose of medication.
7. Significant hepatic impairment
8. Significant renal impairment
9. History of porphyria
10. History of leukemia or other malignancy
11. Participation in a clinical drug trial within 4 weeks
12. Severe sleep apnea or excess daytime sleepiness
13. Recent or anticipated use of prohibited medications
14. Have any other condition which, in the Investigator’s opinion, would put the person with Down syndrome at risk by participating in this study
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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. <br>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.[At 2 weeks, 4 weeks, 8 weeks, 12 weeks and 16 weeks]
- Secondary Outcome Measures
Name Time Method 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)]. [At 4 weeks, 12 weeks, 16 weeks];Effects of BTD-001 on auditory event related potentials (ERP) in persons with Down syndrome. Auditory ERP will be assessed by electroencephalography (EEG).[At 4 weeks, 12 weeks, 16 weeks]