None listed
Conditions
Brief summary
BTD-001, has been used for several decades around the world as a treatment for dementia, a respiratory stimulant and as an ingredient of a cough medication. Studies in a mouse model of Down syndrome (DS) (Ts65Dn transgenic mouse) have demonstrated that chronic administration of the product improved cognition on a number of behavioural assessments. These improvements were sustained months after discontinuation of drug administration, indicating that BTD-001 may cause long-lasting synaptic changes supporting improved cognition. These data indicate that BTD-001, may improve function and cognition in persons with DS and has the potential to improve educational and quality of life outcomes for this population.
Interventions
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 s
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.
Sponsors
Novotech (Australia) Pty Limited
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used)
Eligibility
Sex/Gender
All
Age
13 Years to 35 Years
Healthy volunteers
No
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 status.
Exclusion criteria
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
Outcome results
None listed