Tasimelteon in blind children with non-24hrs sleep-wake disorder
Research type
Research Study
Full title
OPEN-LABEL STUDY TO INVESTIGATE THE PHARMACOKINETICS AND SAFETY OF TASIMELTEON IN TOTALLY BLIND CHILDREN AND ADOLESCENTS WITH NON-24-HOUR SLEEP- WAKE DISORDER
IRAS ID
190186
Contact name
Dr Catherine M Hill
Contact email
Sponsor organisation
Vanda Pharmaceuitcals Inc.
Eudract number
2015-003394-15
Clinicaltrials.gov Identifier
U.S. IND Number:, 54,776
Duration of Study in the UK
1 years, 10 months, 12 days
Research summary
Non-24 Sleep-Wake disorder (Non-24) is a circadian rhythm disorder where a person’s internal body clock or circadian clock is not aligned with the 24-hour day, (everyone’s internal body clock runs a little longer than 24 hours),for sighted people, it is reset every day by environmental light cues. For people who are unable to perceive light, the body clock can become out of sync with the 24-hour day-night cycle. Up to 70% of totally blind individuals suffer from Non-24. The main symptoms of Non-24 are difficulty falling asleep , waking up in the morning, or staying awake during the day.
Tasimelteon is an agonist – a substance that binds to the melatonin receptors MT1 and MT2, thought to be involved in the control of the body clock. This drug has been approved for use in adults but it is believed that Non-24 is present in children with total blindness. Currently, there is no approved treatment for Non-24 in children and adolescents.
Vanda Pharmaceuticals is sponsoring a study to collect information in totally blind children and adolescents from 3-17 years on the safety and effectiveness of tasimelteon.
Approximately 24 participants with a confirmed diagnosis of Non-24 will be enrolled onto the study.
Group 1: Children from 3 to less than 6 years of age (6 participant)
Group 2: Children from 6 to less than 12 years of age (6 participants)
Group 3: Adolescents from 12 to less than 18 years of age (12 participant)
Participants will have their sleep wake cycles assessed in detail and once diagnosis is confirmed, will receive a dose of tasimelteon based on their weight.
This is an open label study, to measure pharmacokinetics and safety on a single day (single dose). There is no placebo (drug that has no medical effect) in this study. This multicentre study will take place at around 5 centres in Europe and the USA, with a target of 3 participants in the UK.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
16/NE/0317
Date of REC Opinion
17 Nov 2016
REC opinion
Further Information Favourable Opinion