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: 19.0 Level: PT Classification code 10040984 Term: Sleep disorder System Organ Class: 10037175 - Psychiatric disorders
Conditions
Interventions
Product Name: Tasimelteon
Product Code: VEC-162
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Tasimelteon
CAS Number: 609799-22-06
Current Sponsor code: VEC-162
Other descriptive name: TAS
Sponsors
Vanda Pharmaceuitcals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To determine a dose for pediatric subjects for which systemic exposure (AUC) is centered around the value expected in adults based on the nominal 20 mg adult dose. • To characterize the pharmacokinetics of an age-appropriate oral formulation of tasimelteon after a single dose administration. • To characterize metabolites M9, M11, M12, M13, and M14 pharmacokinetics in children and adolescents after a single dose administration of an age-appropriate oral formulation of tasimelteon. • To determine the safety and tolerability of a single oral dose of an age-appropriate oral formulation of tasimelteon. ;Secondary Objective: Not applicable;Primary end point(s): Pharmacokinetics Endpoints: • Individual subject plasma concentration for tasimelteon and its metabolites with actual blood sampling times. Due to the sparseness of data, the analysis will not attempt to predict a new set of pharmacokinetic parameters for these subjects. This information will be used to determine an optimal model for pediatric subjects, based on the pharmacokinetic parameters in adults, with appropriate scaling for body size. If the PK analysis of the pediatric population shows that the PK profile in children is different to the adult population, the sampling times above will be modified to ensure the optical design for sampling.;Timepoint(s) of evaluation of this end point: Individual subject plasma concentration for tasimelteon, actual blood sampling times, and pharmacokinetic parameters will be listed. Plasma concentrations and pharmacokinetic parameters will be summarized using descriptive statistics. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety Endpoints: • Safety and tolerability of a single age-appropriate dose of tasimelteon will be assessed as follows: subjective tolerability from spontaneous reporting of Adverse Events (AEs) and AEs collected through the Pediatric Adverse Event Reporting System (PAERS), changes in clinical laboratory parameters that are relevant to safety and influence of trial medication on vital signs and ECG parameters. • The Columbia-Suicide Severity Scale (C-SSRS) will be used in children > 6 years to assess suicidal behavior and ideation. ;Timepoint(s) of evaluation of this end point: All AEs recorded during the study will be listed and tabulated by body system and preferred term. Descriptive statistics will be provided for background and demographic variables such as age, weight, height, gender, pubertal stage, and race. The statistical analyses will be detailed in the Statistical Analysis Plan (SAP). | — |
Countries
France, Germany, United Kingdom, United States
Contacts
Public ContactJoseph Hull
Vanda Pharmaceuitcals Inc.
Outcome results
None listed