Pitolisant for Idiopathic Hypersomnia

Recruiting at 68 trial locations

Overseen By Edward Mezerhane, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Harmony Biosciences Management, Inc.

Must not be taking: QT prolonging drugs

Disqualifiers: Breastfeeding, ESRD, Severe hepatic impairment, 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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

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

Compensation: Varies

Number of Visits: 21

128 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing the safety and effectiveness of pitolisant, a medication that helps people stay awake, in adults with idiopathic hypersomnia. These patients have already completed an earlier part of the study. Pitolisant works by boosting brain chemicals that make you feel more alert. It has been approved for the treatment of narcolepsy in adults.

Will I have to stop taking my current medications?

The trial requires participants to stop taking any medications that are specifically prohibited in the study protocol. If you are taking a medication known to prolong the QT interval, you must discontinue it to participate.

What data supports the effectiveness of the drug Pitolisant for Idiopathic Hypersomnia?

Pitolisant has been shown to help reduce excessive daytime sleepiness in people with narcolepsy, a condition similar to idiopathic hypersomnia, by increasing wakefulness through its action on histamine receptors in the brain. It has also been used successfully in patients with sleepiness that doesn't respond to other stimulants. ^{1 2 3 4 5}

Is Pitolisant safe for humans?

Pitolisant, also known as Wakix, has been used to treat narcolepsy and is generally well tolerated in clinical trials. Common side effects include headache, insomnia (trouble sleeping), nausea, and anxiety, but it has a minimal risk of abuse and is not a controlled substance in the USA. ^{1 3 4 5 6}

How is the drug Pitolisant unique for treating idiopathic hypersomnia?

Pitolisant is unique because it works by increasing histamine release in the brain, which helps promote wakefulness, and it is the only drug of its kind not classified as a controlled substance in the USA, making it a novel option for those who do not respond to other stimulants. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults with idiopathic hypersomnia who finished the prior study phase (HBS-101-CL-010) and can follow the study plan. Participants must not use certain drugs, have severe kidney or liver issues, or be at significant suicide risk. Women of childbearing age must test negative for pregnancy and agree to avoid pregnancy during the study.

Inclusion Criteria

Completed the Double-Blind Randomized Withdrawal Phase (EOT/Visit 5) from the HBS-101-CL-010 study

Is able to provide voluntary, informed consent

I understand and can follow the study's requirements.

Exclusion Criteria

Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to an unstable or uncontrolled medical condition or one that might interfere with the conduct of the study, confound interpretation of study results, pose a health risk to the patient, or compromise the integrity of the study

Does not agree to discontinue any prohibited medication or substances listed in the protocol

Participation in an interventional research study with an investigational medication or device, other than pitolisant, for the duration of the study

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Titration

Participants are titrated up to a maximum dose of 35.6 mg of pitolisant over a 3-week period

3 weeks

Weekly visits for dose titration

Long-Term Dosing

Participants receive long-term dosing of pitolisant with dose adjustments based on safety and effectiveness

Up to approximately 3 years

On-site visits every 6 months, monthly telephone contacts

Follow-up

Participants receive safety follow-up telephone contacts to assess for adverse events and concomitant medication use

1 month

Telephone contacts at 15 and 30 days post-final dose

Treatment Details

Interventions

• Pitolisant

Trial Overview The trial is evaluating the long-term safety and effectiveness of a medication called pitolisant in patients with excessive daytime sleepiness due to idiopathic hypersomnia, following their participation in an earlier phase of research.

Participant Groups

Group I: Pitolisant Experimental Treatment 1 Intervention

Week 1: 8.9 mg pitolisant administered once daily in the morning upon wakening; Week 2: 17.8 mg pitolisant administered once daily in the morning upon wakening; Weeks 3 through end of treatment: 17.8 mg to 35.6 mg pitolisant administered once daily in the morning upon wakening.

Pitolisant is already approved in European Union, United States for the following indications:

Approved in European Union as Wakix for:

• Narcolepsy with or without cataplexy

Approved in United States as Wakix for:

• Excessive daytime sleepiness (EDS) associated with narcolepsy
• Cataplexy in adults with narcolepsy

Who Is Running the Clinical Trial?

Harmony Biosciences Management, Inc.

Lead Sponsor

Trials

9

Recruited

2,000+

Harmony Biosciences, LLC

Lead Sponsor

Trials

9

Recruited

2,000+

Findings from Research

Pitolisant (Wakix™) is an approved treatment in the EU for narcolepsy, effectively reducing excessive daytime sleepiness and cataplexy in adults, as demonstrated in the pivotal HARMONY I trial.

The drug works as an inverse agonist of the histamine H3 receptor and has received special designations, including orphan drug status, highlighting its importance for patients with narcolepsy who have not responded to other treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pitolisant: First Global Approval. Syed, YY.[2019]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Effects of pitolisant, a histamine H3 inverse agonist, in drug-resistant idiopathic and symptomatic hypersomnia: a chart review. [2019]

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

Pitolisant for Residual Excessive Daytime Sleepiness in OSA Patients Adhering to CPAP: A Randomized Trial. [2021]

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

Pitolisant: First Global Approval. [2019]

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

Pitolisant for treating patients with narcolepsy. [2020]

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

Pitolisant: A Review in Narcolepsy with or without Cataplexy. [2021]

6. Germany pubmed.ncbi.nlm.nih.gov

Pitolisant for the treatment of narcolepsy with or without cataplexy. [2019]