NCT02231008

Brief Summary

The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
49

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2015

Longer than P75 for phase_2

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 29, 2014

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 3, 2014

Completed
12 months until next milestone

Study Start

First participant enrolled

September 1, 2015

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 19, 2018

Completed
3.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2022

Completed
Last Updated

November 18, 2022

Status Verified

November 1, 2022

Enrollment Period

3.2 years

First QC Date

August 29, 2014

Last Update Submit

November 15, 2022

Conditions

Smith-Magenis SyndromeCircadian

Keywords

SMS

Outcome Measures

Primary Outcomes (1)

  • To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters

    9 Weeks

Secondary Outcomes (1)

  • Safety and tolerability as measured by spontaneous reporting of adverse events (AEs).

    up to 137 weeks

Study Arms (2)

Tasimelteon

EXPERIMENTAL

Single dose oral capsule or equivalent age-appropriate oral formulation, daily dosage

Drug: tasimelteon

Placebo

PLACEBO COMPARATOR

Placebo comparator

Drug: placebo

Interventions

tasimelteonDRUG
Tasimelteon
placeboDRUG
Placebo

Eligibility Criteria

Age3 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Unknown Facility

Santa Monica, California, United States

Location

Unknown Facility

Baltimore, Maryland, United States

Location

Unknown Facility

Chevy Chase, Maryland, United States

Location

Unknown Facility

Houston, Texas, United States

Location

MeSH Terms

Conditions

Smith-Magenis Syndrome

Interventions

tasimelteon

Condition Hierarchy (Ancestors)

Chronobiology DisordersNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, Inborn

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 29, 2014

First Posted

September 3, 2014

Study Start

September 1, 2015

Primary Completion

November 19, 2018

Study Completion

January 1, 2022

Last Updated

November 18, 2022

Record last verified: 2022-11

Locations

US(4)