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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called tasimelteon to determine its effectiveness in addressing sleep problems in people with Autism Spectrum Disorder (ASD). The goal is to assess whether taking tasimelteon once a day is safe and improves sleep. It is open to individuals diagnosed with ASD who experience sleep difficulties not linked to other conditions or medications. Participants must be willing to follow the study rules and should not have liver issues or a history of not tolerating tasimelteon. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for sleep issues in ASD.

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.

Is there any evidence suggesting that tasimelteon is likely to be safe for humans?

Research shows that tasimelteon is generally safe for adults. The FDA has approved it for treating a specific sleep disorder, indicating its safety for certain uses. However, its safety for treating sleep disorders in children under 3 years old, like those in this study, remains uncertain.

Past studies have used tasimelteon to address sleep problems without major safety issues. Nonetheless, side effects such as headaches or drowsiness can occur. As with any medication, monitoring its effects on each individual is crucial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism and sleep disorders?

Tasimelteon is unique because it targets the body’s circadian rhythms, which are often disrupted in individuals with autism and sleep disorders. Unlike other treatments that mainly focus on sedating the patient, Tasimelteon acts on melatonin receptors to help reset the body's internal clock. Researchers are excited because this approach could improve sleep quality and consistency without the side effects commonly associated with traditional sleep medications.

What evidence suggests that tasimelteon might be an effective treatment for sleep disorders in autism?

Research has shown that tasimelteon, which participants in this trial will receive, can help with nighttime sleep problems. It improved sleep in people with Smith-Magenis syndrome, a condition often linked to sleep issues. Tasimelteon mimics melatonin, a natural hormone that regulates sleep patterns. Early results suggest that tasimelteon can enhance sleep from the first night of use, with benefits persisting over time. Although limited data exist on its effects for people with autism spectrum disorder (ASD), its success in treating other sleep problems suggests it might also benefit those with ASD. ^{2 4 6 7 8}

Are You a Good Fit for This Trial?

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.

Evidence of increased risk of self-harm

Pregnant or lactating females

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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, 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]

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 (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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05361707

NCT05361707 | Evaluating the Effects of Tasimelteon in ... This is a multicenter, open-label study to evaluate the efficacy and safety of a daily single oral dose of tasimelteon in treating sleep disturbances in ...

2. trial.medpath.com trial.medpath.com/drug/e6cd1999923d084d/tasimelteon

Tasimelteon | MedPath Tasimelteon oral suspension is indicated for the treatment of nighttime sleep disturbances in Smith-Magenis syndrome in patients 3 to 15 years of age.

3. nhpri.org nhpri.org/blobnhpri08e0944faa/wp-content/uploads/2024/06/tasimelteon-Hetlioz-Hetlioz-LQ-2426-A-SGM-P2024.pdf

tasimelteon-Hetlioz-Hetlioz-LQ-2426-A-SGM-P2024. ... For nighttime sleep disturbances in Smith-Magenis syndrome, both of the following: i. Chart notes or test results confirming Smith-Magenis Syndrome. ii.

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40971945/

Melatonin agonist tasimelteon (HETLIOZ®) improves sleep ... Conclusion: Tasimelteon improved sleep from the first night of treatment, and the effect continued for the duration of the study. Tasimelteon ...

5. withpower.com withpower.com/trial/phase-3-sleep-wake-disorders-6-2021-4d24b

Tasimelteon for Autism and Sleep Disorders This trial is testing a daily pill called tasimelteon to help children and adults with Autism Spectrum Disorder (ASD) who have trouble sleeping.

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2020/214517s000lbl.pdf

Hetlioz (tasimelteon) - accessdata.fda.gov Safety and effectiveness of HETLIOZ for the treatment of nighttime sleep disturbances in SMS have not been established in patients younger than 3 years old.

7. hetlioz.com hetlioz.com/

HETLIOZ® (tasimelteon) is the first prescription medication ... The safety and effectiveness of HETLIOZ LQ® oral suspension for the treatment of nighttime sleep disturbances in SMS have been established in pediatric patients ...

8. go.drugbank.com go.drugbank.com/drugs/DB09071

Tasimelteon: Uses, Interactions, Mechanism of Action Tasimelteon oral capsules are indicated for the treatment of non-24 hour sleep-wake disorder in adult patients and for the treatment of nighttime sleep ...