Study of the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents

Open-label Study to Investigate the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents

Open-label Study to Investigate the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder; Smith-Magenis Syndrome; Non-24 Hour Sleep-Wake Disorder; Circadian Rhythm Sleep Disorders
• Intervention / Treatment: Drug: tasimelteon

Detailed Description

This study is an open-label, single dose, non-controlled study to evaluate the PK and safety of tasimelteon in children and adolescents from 3 years to less than 18 years of age who were legally blind and met the diagnostic criteria for CRSWD Non-24 per DSM-V or who were diagnosed with a Neurodevelopmental Disorder (like ASD and SMS) and had a nighttime sleep complaint.

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21225
      • PAREXEL Early Phase Clinical Unit

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Males or females 3 to <18 years of age who are legally blind [defined as having a visual acuity of 20/200 or less in the better-seeing eye with best conventional correction (glasses or contact lenses) and/or a visual field of 20 degrees or less in the better-seeing eye], males or females 3 to <18 years of age with SMS and with a nighttime sleep complaint and males or females 3 to <18 years of age with ASD and with a nighttime sleep complaint;
2. Weigh at least 10 kg;

3 Diagnosis of SMS determined by a prior positive genetic test result as indicated by parent/guardian; Diagnosis of ASD as indicated by parent/guardian; or a diagnosis of Non-24 as determined by DSM-5 diagnostic criteria for the Circadian rhythm sleep-wake disorder, Non-24-hour sleep-wake hour type:

1. A persistent or recurrent pattern of sleep disruption that is primarily due to an alteration of the circadian system or to a misalignment between the endogenous circadian rhythm and the sleep-wake schedule required by an individual's physical environment or social or professional schedule;
2. The sleep disruption leads to excessive sleepiness or insomnia, or both;
3. The sleep disturbance causes clinically significant distress or impairment in social, occupational, and other important areas of functioning.

Exclusion Criteria:

1. For blind subjects only: Subjects who have a probable diagnosis of a current sleep disorder other than Non-24-Hour Sleep-Wake Disorder that is the primary cause of the sleep disturbance based on clinical investigator medical judgment;
2. For blind subjects only: History (within the 12 months prior to screening) of psychiatric disorders including ADHD, Neurodisabilities, Major Depressive Disorder, Generalized Anxiety Disorder, Axis II Disorders, delirium or any other psychiatric disorder, that is not being successfully treated or has not been resolved and that in the opinion of the clinical investigator would affect participation in the study or full compliance with study procedures;
3. History of intolerance and/or hypersensitivity to melatonin or melatonin agonists;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pharmacokinetic Dosing; Single-dose pharmacokinetics of tasimelteon	Drug: tasimelteon; Melatonin receptor agonist; Other Names: Hetlioz

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Area under the curver (AUC) of tasimelteon and its metabolites	Pre-dose, 15 minutes post dose, 30 minutes post dose,1 hour post dose, 2 hours post dose, 4 hours post dose
Maximum concetration (Cmax) of tasimelteon and its metabolites	Pre-dose, 15 minutes post dose, 30 minutes post dose,1 hour post dose, 2 hours post dose, 4 hours post dose
Steady-state concentration (Css) of tasimelteon and its metabolites	Pre-dose, 15 minutes post dose, 30 minutes post dose,1 hour post dose, 2 hours post dose, 4 hours post dose
Half-life of tasimelteon and its metabolites	Pre-dose, 15 minutes post dose, 30 minutes post dose,1 hour post dose, 2 hours post dose, 4 hours post dose
Trough concentration (Ctrough) of tasimelteon and its metabolites	Pre-dose, 15 minutes post dose, 30 minutes post dose,1 hour post dose, 2 hours post dose, 4 hours post dose
Safety and tolerability of tasimelteon as measured by spontaneous reporting of adverse events (AEs)	Day 1
Safety and tolerability of tasimelteon as measured by Pediatric Adverse Event Reporting System (PAERS)	Day 1

Collaborators and Investigators

• Sponsor: Vanda Pharmaceuticals
• Investigators: Study Director: Vanda Pharmaceuticals, Sponsor GmbH

Study record dates

Study Major Dates

• Study Start (Actual): October 4, 2016
• Primary Completion (Actual): December 20, 2017
• Study Completion (Actual): December 20, 2017

Study Registration Dates

• First Submitted: May 16, 2016
• First Submitted That Met QC Criteria: May 17, 2016
• First Posted (Estimated): May 18, 2016

Study Record Updates

• Last Update Posted (Actual): March 21, 2024
• Last Update Submitted That Met QC Criteria: March 19, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Dyssomnias; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Occupational Diseases; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Abnormalities, Multiple; Chromosome Disorders; Chronobiology Disorders; Sleep Wake Disorders; Autism Spectrum Disorder; Sleep Disorders, Circadian Rhythm; Smith-Magenis Syndrome
• Other Study ID Numbers: VP-VEC-162-4201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO