Circadian, Smith-Magenis Syndrome
Conditions
Keywords
SMS
Brief summary
The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome.
Interventions
DRUGtasimelteon
DRUGplacebo
Sponsors
Vanda Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
3 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. A confirmed clinical diagnosis of SMS 2. Informed consent from the patient or the legal guardian 3. Male or female between the ages of 3- 65 years of age 4. Recent history of sleep disturbances 5. Have an appointed care-giver complete the required outpatient assessments 6. Willing and able to comply with study requirements and restrictions
Exclusion criteria
1. Unable to dose daily with medication 2. Exposure to any investigational drug, including placebo, within 30 days or 5 half-lives (whichever was longer) of screening 3. Any other sound medical reason as determined by the clinical investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters | 9 Weeks |
Secondary
| Measure | Time frame |
|---|---|
| Safety and tolerability as measured by spontaneous reporting of adverse events (AEs). | up to 137 weeks |
Countries
United States
Outcome results
None listed