Tasimelteon for Autism and Sleep Disorders

Recruiting at 4 trial locations

Overseen By Vanda Pharmaceuticals Inc.

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Vanda Pharmaceuticals

Must not be taking: Sedatives, Stimulants

Disqualifiers: Impaired liver function, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

100 Participants Needed

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. ^{1 2 3 4 5}

Is Tasimelteon safe for humans?

Tasimelteon has been generally well tolerated in studies, with common mild side effects like headache, unusual dreams, and minor infections. No serious adverse events or deaths were reported, and it is considered safe for long-term use in treating sleep disorders. ^{1 2 3 4 6}

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. ^{1 2 3 6 7}

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.

Exclusion Criteria

My liver isn't working properly.

Pregnant or lactating females

Evidence of increased risk of self-harm

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

Trial Overview The 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

Group I: Tasimelteon Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Vanda Pharmaceuticals

Lead Sponsor

Trials

68

Recruited

19,900+

Findings from Research

In two phase III trials involving totally blind individuals with Non-24-Hour Sleep-Wake Disorder, tasimelteon (20 mg once nightly) significantly improved the ability to entrain the circadian pacemaker compared to placebo.

Tasimelteon was generally well tolerated and also led to improvements in sleep/wake parameters and overall functioning, making it a valuable treatment option for Non-24 in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tasimelteon: A Review in Non-24-Hour Sleep-Wake Disorder in Totally Blind Individuals. Keating, GM.[2018]

Tasimelteon (Hetlioz™) is a melatonin-receptor agonist approved by the FDA in January 2014 specifically for treating non-24-hour sleep-wake disorder, indicating its targeted therapeutic use.

The article reviews tasimelteon's mechanism of action, pharmacokinetics, and its clinical efficacy, safety, and tolerability, highlighting its potential benefits for individuals with this specific sleep disorder.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tasimelteon (Hetlioz™): A New Melatonin Receptor Agonist for the Treatment of Non-24-Hour Sleep-Wake Disorder. Bonacci, JM., Venci, JV., Gandhi, MA.[2015]

Tasimelteon (Hetlioz®) is the first FDA-approved treatment for non-24-hour sleep-wake disorder, particularly effective in blind individuals, as it helps reduce nighttime wakefulness and daytime sleepiness by promoting synchronization to the 24-hour cycle.

The drug is well tolerated with common side effects including headaches and unusual dreams, and ongoing safety studies are being conducted to further assess its effects in blind patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tasimelteon for the treatment of non-24-hour sleep-wake disorder. Neubauer, DN.[2017]

Tasimelteon is an effective treatment for non-24 sleep-wake rhythm disorder, helping to resynchronize the circadian rhythms of individuals who are totally blind, with significant improvements in sleep-wake functions.

Long-term use of tasimelteon is safe and well tolerated, and it is the only FDA and EMA approved medication specifically for non-24, with studies showing that 90% of patients who continued treatment maintained circadian entrainment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tasimelteon for treating non-24-h sleep-wake rhythm disorder. Nishimon, S., Nishimon, M., Nishino, S.[2019]

Tasimelteon, a melatonin receptor agonist, is the first approved treatment for Non-24-hour Sleep-Wake Disorder, showing a strong affinity for the MT2 receptor, which helps regulate sleep cycles.

In a study involving 184 blind individuals over more than a year, tasimelteon was found to be safe and well-tolerated, with adverse effects similar to those seen in placebo groups, indicating its potential for long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety profile of tasimelteon, a melatonin MT1 and MT2 receptor agonist: pooled safety analyses from six clinical studies. Leger, D., Quera-Salva, MA., Vecchierini, MF., et al.[2015]

Tasimelteon, a dual melatonin receptor agonist approved for Non-24-Hour Sleep-Wake Disorder, showed an absolute bioavailability of 38% in a study involving 14 healthy volunteers, indicating effective absorption when taken orally.

The study found that tasimelteon was well tolerated, with only mild treatment-emergent adverse events reported in 21.4% of participants, and no serious adverse events or discontinuations, highlighting its safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Absolute Bioavailability of Tasimelteon. Torres, R., Dressman, MA., Kramer, WG., et al.[2015]

Tasimelteon, the first approved treatment for Non-24-Hour Sleep-Wake Disorder, is a potent Dual Melatonin Receptor Agonist with significantly higher affinity for the MT2 receptor, which is important for regulating circadian rhythms.

In extensive testing, tasimelteon showed minimal interaction with over 160 other receptors and enzymes, suggesting a favorable safety profile and specificity for its intended action.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tasimelteon: a selective and unique receptor binding profile. Lavedan, C., Forsberg, M., Gentile, AJ.[2015]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Tasimelteon: A Review in Non-24-Hour Sleep-Wake Disorder in Totally Blind Individuals. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Tasimelteon (Hetlioz™): A New Melatonin Receptor Agonist for the Treatment of Non-24-Hour Sleep-Wake Disorder. [2015]

3. Spain pubmed.ncbi.nlm.nih.gov

Tasimelteon for the treatment of non-24-hour sleep-wake disorder. [2017]

4. England pubmed.ncbi.nlm.nih.gov

Tasimelteon for treating non-24-h sleep-wake rhythm disorder. [2019]

5. England pubmed.ncbi.nlm.nih.gov

Safety profile of tasimelteon, a melatonin MT1 and MT2 receptor agonist: pooled safety analyses from six clinical studies. [2015]

6. United States pubmed.ncbi.nlm.nih.gov

Absolute Bioavailability of Tasimelteon. [2015]

7. England pubmed.ncbi.nlm.nih.gov

Tasimelteon: a selective and unique receptor binding profile. [2015]