OPEN-LABEL STUDY TO INVESTIGATE THE PHARMACOKINETICS AND SAFETY OF TASIMELTEON IN TOTALLY BLIND CHILDREN AND ADOLESCENTS WITH NON-24-HOUR SLEEP- WAKE DISORDER

Phase 1

• Conditions: This study will be an open-label, single dose, non-controlled trial to evaluate the pharmacokinetics and safety of tasimelteon in children who are 3 years to less than 18 years of age, totally blind with no conscious light perception and exhibit/have Non-24-Hour Sleep-Wake Disorder; MedDRA version: 19.0Level: PTClassification code 10040984Term: Sleep disorderSystem Organ Class: 10037175 - Psychiatric disorders; Therapeutic area: Not possible to specify

• Registration Number: EUCTR2015-003394-15-DE
• Lead Sponsor: Vanda Pharmaceuitcals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-07-25
• Last Update Posted: 9 January 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

1. Written informed consent from parents having parental responsibility or from the legal guardian(s). In the case of a child is aged 12 years or older the written assent of the child is needed in addition to that of parents having responsibility/legal guardian;

2. Males or females between 3 to <18 years;

3. Children in cohort 1 (age 3 to < 6) must be daytime toilet trained;

4. Body Mass Index (BMI) within the 5th and 95th percentile based on age (BMI =
weight (kg)/ [height (m)]2);

5. Weigh at least 16 kg;

6. Post-pubescent girls already having menses must have a negative pregnancy test at the screening and baseline visits;

7. No perception of light and Diagnosis of Non-24-Hour Sleep-Wake Disorder as determined by:

a.History (within the last 3 months) of trouble sleeping at night (difficulty initiating sleep or staying asleep), difficulty awakening in the morning, or daytime sleepiness as determined by answering yes to at least one question in the Sleep Complaint Questionnaire, and

b. A non-entrained urinary aMT6s rhythm defined as a tau = 24.10 h and 95% confidence interval (CI) that does not include 24;

8. Willing and able to comply with study requirements and restrictions including a commitment to a fixed sleep opportunity during the study;

9. Subjects who fail to respond to behavior modification/sleep hygiene measures during the Pre-Enrollment Phase as determined by the physician through patient interview and/or sleep diaries

Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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. History (within the 12 months prior to screening) of psychiatric disorders including ADHD, Autism, 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;

4. Subjects having any suicidal ideation of type 4 or 5 on the C-SSRS at Screening or Baseline;

5. Subject is at risk of suicide, in the opinion of the Investigator. Evidence of suicide risk could include any suicide attempt within the past year or any other suicidal behavior within the past year;

6. Current clinically significant cardiovascular, respiratory, neurologic, hepatic, hematopoietic, renal, gastrointestinal or metabolic dysfunction unless currently controlled and stable;

7. Clinically significant deviation from normal in clinical laboratory results, vital signs measurements, or physical examination findings at screening as determined by the clinical investigator;

8. Indication of impaired liver function (values for AST, ALT or bilirubin > 2 times
Upper Limit of Normal);

9. Pregnant or lactating females;

10. Smokers;

11. Exposure to any investigational drug, including placebo, within 30 days or 5 half- lives (whichever was longer) of screening;

12. Unwilling or unable to follow the medication restrictions described in Section 8.2, or unwilling or unable to sufficiently wash-out from use of a restricted medication;

13. Any other sound medical reason as determined by the clinical investigator

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified