Tasimelteon for Autism and Sleep Disorders
Recruiting in Palo Alto (17 mi)
+4 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Vanda Pharmaceuticals
Must not be taking: Sedatives, Stimulants
Disqualifiers: Impaired liver function, Pregnancy, others
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 2 Jurisdictions
Trial Summary
What is the purpose of this trial?
This trial is testing a daily pill called tasimelteon to help children and adults with Autism Spectrum Disorder (ASD) who have trouble sleeping. The medication aims to improve sleep by helping to regulate the body's internal clock.
Will I have to stop taking my current medications?
The trial requires that participants do not use medications that may cause sedation or stimulation, so you might need to stop taking such medications.
What data supports the effectiveness of the drug Tasimelteon for treating sleep disorders in autism?
Tasimelteon has been shown to help regulate sleep patterns in people with Non-24-Hour Sleep-Wake Disorder, especially in totally blind individuals, by aligning their sleep-wake cycles with the 24-hour day. This suggests it might also help with sleep issues in autism, as it works by targeting melatonin receptors to improve sleep-wake functions.12345
Is Tasimelteon safe for humans?
How is the drug Tasimelteon unique for treating autism and sleep disorders?
Tasimelteon is unique because it is a dual melatonin receptor agonist, specifically targeting the MT2 receptor to help regulate the body's internal clock, which is different from other treatments that may not focus on circadian rhythm adjustment. It is also the first drug approved for Non-24-Hour Sleep-Wake Disorder, highlighting its novel approach to sleep regulation.12367
Eligibility Criteria
This trial is for children and adults aged 2 to 65 with Autism Spectrum Disorder (ASD) who have trouble sleeping. Participants must be able to follow the study's rules and give consent, or have a guardian who can consent for them. They shouldn't have liver problems, drug abuse issues, or other sleep disorders caused by different conditions.Inclusion Criteria
Ability and acceptance to provide written informed consent of the participant or legal guardian (and assent as required)
Willing and able to comply with study requirements and restrictions
I have been diagnosed with ASD and have recent trouble sleeping.
See 2 more
Exclusion Criteria
My liver isn't working properly.
Pregnant or lactating females
Evidence of increased risk of self-harm
See 3 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive a daily single oral dose of tasimelteon to treat sleep disturbances
12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Treatment Details
Interventions
- Tasimelteon (Melatonin Receptor Agonist)
Trial OverviewThe trial is testing how safe and effective tasimelteon, taken as an oral capsule or liquid suspension once daily, is at improving sleep in people with ASD. It's an open-label study which means everyone knows they're getting the actual medication.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: TasimelteonExperimental Treatment1 Intervention
Drug: Tasimelteon
Tasimelteon is already approved in United States, European Union for the following indications:
🇺🇸 Approved in United States as Hetlioz for:
- Non-24-Hour Sleep-Wake Disorder
- Nighttime sleep disturbances in Smith-Magenis Syndrome
🇪🇺 Approved in European Union as Hetlioz for:
- Non-24-hour sleep-wake rhythm disorder in totally blind adults
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Vanda Investigational SiteStaten Island, NY
Vanda Investigational SiteBoulder, CO
Vanda Investigational SiteSanta Monica, CA
Vanda Investigational SiteSan Jose, CA
More Trial Locations
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Who Is Running the Clinical Trial?
Vanda PharmaceuticalsLead Sponsor
References
Tasimelteon: A Review in Non-24-Hour Sleep-Wake Disorder in Totally Blind Individuals. [2018]Tasimelteon (Hetlioz(®)) is a dual melatonin receptor agonist indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (Non-24) (free-running disorder). In two randomized, double-masked, multicentre, phase III trials, totally blind individuals with Non-24 who received oral tasimelteon 20 mg once nightly were significantly more likely than those receiving placebo to entrain the circadian pacemaker (the SET trial) and maintain entrainment (the RESET trial). Sleep/wake parameters and functioning were also improved with tasimelteon. Oral tasimelteon was generally well tolerated in totally blind patients with Non-24. In conclusion, tasimelteon is a useful drug for the treatment of Non-24 in totally blind individuals.
Tasimelteon (Hetlioz™): A New Melatonin Receptor Agonist for the Treatment of Non-24-Hour Sleep-Wake Disorder. [2015]In January 2014, the US Food and Drug Administration approved tasimelteon (Hetlioz™), a melatonin-receptor agonist for the treatment of non-24-hour sleep-wake disorder. This article provides an overview of the mechanism of action, pharmacokinetic properties, as well as the clinical efficacy, safety, and tolerability of tasimelteon. Relevant information was identified through a comprehensive literature search of several databases using the key words tasimelteon, Non-24-hour Sleep-Wake disorder, Non-24, and melatonin. Further information was obtained from the tasimelteon package insert, fda.gov, clinicaltrials.gov, briefing materials provided by Vanda Pharmaceuticals, and posters from scientific meetings.
Tasimelteon for the treatment of non-24-hour sleep-wake disorder. [2017]Tasimelteon (Hetlioz®), a melatonin receptor agonist, is the first, and, at the time of the publication, the only drug to be approved by the U.S. Food and Drug Administration (FDA) for the treatment of non-24-hour sleep-wake disorder (non-24). This circadian rhythm disorder occurs most commonly in blind individuals without light perception, and it results from their inability to entrain to the 24-hour photoperiod, although the indication does not specify a particular patient population. Non-24 is characterized by a persistent cycle of nighttime insomnia and daytime sleepiness, alternating with asymptomatic periods depending on an individual's degree of circadian rhythm synchronization with the photoperiod at any particular time. Phase II clinical trials in healthy individuals confirmed the circadian phase-shifting potential of tasimelteon. Phase III trials in totally blind subjects diagnosed with non-24 demonstrated the efficacy of tasimelteon in reducing both nighttime wakefulness and daytime napping. Physiologic monitoring revealed that tasimelteon resulted in a higher proportion of individuals becoming entrained to the 24-hour cycle compared with placebo. Safety assessments indicated that tasimelteon is well tolerated, with the most common adverse events being headache, alanine aminotransferase elevation, nightmares or unusual dreams, and upper respiratory or urinary tract infections. Tasimelteon is available as a capsule in a single 20-mg dose and it must be obtained through Vanda Pharmaceutical's HetliozSolutions program with dispensing through a specialty pharmacy. Safety studies in blind individuals diagnosed with non-24 are ongoing and a future clinical trial with Smith-Magenis syndrome patients is planned.
Tasimelteon for treating non-24-h sleep-wake rhythm disorder. [2019]Non-24-h sleep-wake rhythm disorder (non-24) is observed in approximately half of totally blind individuals, a condition caused by their circadian pacemaker in the suprachiasmatic nucleus not being entrained to 24 h due to a lack of light perceptions. These subjects have a progressively delayed circadian cycle each day, non-24 periodically inducing nighttime insomnia and daytime sleepiness. Tasimelteon is a dual melatonin receptor agonist with high affinity for the melatonin 2 receptor, and it entrains endogenous melatonin rhythms and sleep-wake cycles in individuals with non-24. Areas covered: Herein, the authors review the treatment of non-24 with tasimelteon. The authors further compare tasimelteon with other melatonin receptor agonists. Expert opinion: The treatment strategies for non-24 aim to resynchronize the free-running rhythm with the 24 h light-dark cycle. Tasimelteon entrains circadian rhythms and improves the sleep-wake functions of individuals with non-24. Furthermore, the RESET study demonstrated that 20% of patients that discontinued tasimelteon maintained circadian entrainment whereas 90% of those who continued it maintained circadian entrainment. Long-term administration of tasimelteon is safe and well tolerated, and it is the only pharmacological therapy approved by the US Food and Drug Administration and the European Medicines Agency for non-24.
Safety profile of tasimelteon, a melatonin MT1 and MT2 receptor agonist: pooled safety analyses from six clinical studies. [2015]Tasimelteon, a novel circadian regulator, is the first product for the treatment of Non-24-hour Sleep-Wake Disorder (Non-24) approved by either the FDA or the European Medicines Agency (EMA). Tasimelteon is a potent and specific melatonin (MT1 and MT2) receptor agonist with 2 - 4 times greater affinity for the MT2 receptor.
Absolute Bioavailability of Tasimelteon. [2015]Tasimelteon is a novel dual melatonin receptor agonist and is the first treatment approved by the US Food and Drug Administration for Non-24-Hour Sleep-Wake Disorder. This study was conducted to assess the absolute bioavailability of tasimelteon and to further assess the single-dose pharmacokinetics, safety, and tolerability of oral and intravenous (IV) routes of administration of the drug. This study was an open-label, single-dose, randomized, 2-period, 2-treatment, 2-sequence, crossover study in which 14 healthy volunteers were randomly administered tasimelteon as either a 20-mg capsule or IV administration of 2 mg infused over 30 minutes. Each subject received both treatments in a random order, separated by a washout period of 5 ± 2 days. The total clearance and volume of distribution of tasimelteon, from the IV treatment, were 505 mL per minute and 42.7 L, respectively. Based on the statistical comparison of dose-corrected area under the curve to infinity, the absolute bioavailability was 38%, with a 90% confidence interval of 27%-54%. The mean elimination half-life was the same for the oral and IV routes. The exposure ratios, oral-to-IV, for metabolites M9, M11, M12, and M13, were 133.27%, 118.28%, 138.76%, and 112.36%, respectively, suggesting presystemic or first-pass metabolism. Three (21.4%) subjects experienced a treatment-emergent adverse event (TEAE) during the study. All TEAEs were mild, considered related to study medication, and consistent with what has been seen in other studies. There were no deaths, serious adverse events, or discontinuations due to TEAEs. Both tasimelteon treatments were well tolerated during the study.
Tasimelteon: a selective and unique receptor binding profile. [2015]Hetlioz(®) (tasimelteon) is the first approved treatment in the United States for Non-24-Hour Sleep-Wake Disorder (Non-24). We present here data on the in vitro binding affinity of tasimelteon for both human melatonin receptors MT1 and MT2, as well as the extended screen of other receptors and enzymes. Results indicate that tasimelteon is a potent Dual Melatonin Receptor Agonist (DMRA) with 2.1-4.4 times greater affinity for the MT2 receptor believed to mediate circadian rhythm phase-shifting (Ki = 0.0692 nM and Ki = 0.17 nM in NIH-3T3 and CHO-K1 cells, respectively), than for the MT1 receptor (Ki = 0.304 nM and Ki = 0.35 nM, respectively). Tasimelteon was also shown to have no appreciable affinity for more than 160 other pharmacologically relevant receptors and several enzymes.