Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS
Primary Purpose
Smith-Magenis Syndrome, Circadian
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
tasimelteon
placebo
Sponsored by
About this trial
This is an interventional treatment trial for Smith-Magenis Syndrome focused on measuring SMS
Eligibility Criteria
Inclusion Criteria:
- A confirmed clinical diagnosis of SMS
- Informed consent from the patient or the legal guardian
- Male or female between the ages of 3- 65 years of age
- Recent history of sleep disturbances
- Have an appointed care-giver complete the required outpatient assessments
- Willing and able to comply with study requirements and restrictions
Exclusion Criteria:
- Unable to dose daily with medication
- Exposure to any investigational drug, including placebo, within 30 days or 5 half-lives (whichever was longer) of screening
- Any other sound medical reason as determined by the clinical investigator
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Tasimelteon
Placebo
Arm Description
Single dose oral capsule or equivalent age-appropriate oral formulation, daily dosage
Placebo comparator
Outcomes
Primary Outcome Measures
To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters
Secondary Outcome Measures
Safety and tolerability as measured by spontaneous reporting of adverse events (AEs).
Full Information
NCT ID
NCT02231008
First Posted
August 29, 2014
Last Updated
November 15, 2022
Sponsor
Vanda Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02231008
Brief Title
Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS
Official Title
A Double-blind, Randomized, Two-period Crossover Study Evaluating the Effects of Tasimelteon vs. Placebo on Sleep Disturbances of Individuals With Smith-Magenis Syndrome (SMS)
Study Type
Interventional
2. Study Status
Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
September 2015 (Actual)
Primary Completion Date
November 19, 2018 (Actual)
Study Completion Date
January 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vanda Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Smith-Magenis Syndrome, Circadian
Keywords
SMS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
49 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Tasimelteon
Arm Type
Experimental
Arm Description
Single dose oral capsule or equivalent age-appropriate oral formulation, daily dosage
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo comparator
Intervention Type
Drug
Intervention Name(s)
tasimelteon
Intervention Type
Drug
Intervention Name(s)
placebo
Primary Outcome Measure Information:
Title
To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters
Time Frame
9 Weeks
Secondary Outcome Measure Information:
Title
Safety and tolerability as measured by spontaneous reporting of adverse events (AEs).
Time Frame
up to 137 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
A confirmed clinical diagnosis of SMS
Informed consent from the patient or the legal guardian
Male or female between the ages of 3- 65 years of age
Recent history of sleep disturbances
Have an appointed care-giver complete the required outpatient assessments
Willing and able to comply with study requirements and restrictions
Exclusion Criteria:
Unable to dose daily with medication
Exposure to any investigational drug, including placebo, within 30 days or 5 half-lives (whichever was longer) of screening
Any other sound medical reason as determined by the clinical investigator
Facility Information:
City
Santa Monica
State/Province
California
Country
United States
City
Baltimore
State/Province
Maryland
Country
United States
City
Chevy Chase
State/Province
Maryland
Country
United States
City
Houston
State/Province
Texas
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS
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