Pitolisant for Prader-Willi Syndrome

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Harmony Biosciences Management, Inc.

Must be taking: Pitolisant

Must not be taking: QT-prolonging medications

Disqualifiers: 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

Compensation: Varies

Number of Visits: 6

Duration: Long-term

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests pitolisant, a medication aimed at improving the health of people with Prader-Willi syndrome, a genetic disorder affecting appetite, growth, and metabolism. The goal is to ensure pitolisant's safety for those who participated in earlier studies of the drug. Individuals who completed a previous pitolisant study are eligible to join. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

Do I have to stop taking my current medications for the trial?

You may need to stop taking certain medications if they are listed as prohibited in the trial protocol. Specifically, you cannot take medications that prolong the QT interval (a heart rhythm measure) during the study.

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

A previous study found that patients generally tolerated pitolisant well. Common side effects included anxiety, irritability, and headache, each affecting about 12% of patients, and these side effects were consistent across different doses. Although pitolisant is being tested specifically for Prader-Willi syndrome (PWS) in this trial, it has already received approval for other conditions, offering some reassurance about its safety. The ongoing Phase 3 study aims to further evaluate its safety for individuals with PWS. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Prader-Willi syndrome?

Pitolisant is unique because it works differently from most treatments for Prader-Willi Syndrome, which typically focus on managing symptoms like obesity and behavioral issues. Unlike these standard options, Pitolisant targets histamine H3 receptors in the brain, which can help improve wakefulness and reduce excessive daytime sleepiness. This new mechanism of action is exciting because it offers a novel way to address some of the neurological symptoms associated with Prader-Willi Syndrome, potentially enhancing quality of life for patients.

What evidence suggests that pitolisant might be an effective treatment for Prader-Willi syndrome?

Research has shown that pitolisant effectively reduces excessive daytime sleepiness in people with Prader-Willi syndrome (PWS). One study found that pitolisant improved alertness and helped with irritability and social withdrawal. Another study discovered it might reduce excessive hunger, a common issue in PWS. These findings suggest that pitolisant could address multiple symptoms in PWS, making it a promising treatment option. ^{1 2 3 4 5}

Who Is on the Research Team?

David Seiden, MD

Principal Investigator

Harmony Biosciences Management, Inc.

Are You a Good Fit for This Trial?

This trial is for individuals with Prader-Willi Syndrome who finished a previous pitolisant study. They must understand and follow the study rules, use contraception if needed, and not be breastfeeding or planning to start. Participants should not have severe liver issues, be at risk of suicide, or be on certain other medications.

Inclusion Criteria

Ability to provide voluntary, written informed consent and, where applicable, voluntary, written assent

Completion of an EOT visit in one of the specified parent studies with pitolisant

I (or my guardian) understand and agree to follow the study's requirements.

Exclusion Criteria

Planning to breastfeed over the course of the study

Not agreeing to discontinue any prohibited medication or substances

Significant risk of committing suicide or suicidality

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pitolisant administered orally once daily in the morning upon awakening

Long-term

Safety assessments every 6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pitolisant

Trial Overview The trial is testing the safety of a drug called pitolisant in people with Prader-Willi Syndrome who've already taken it in earlier studies. It's a continuation to see how they do over a longer period.

How Is the Trial Designed?

Group I: Pitolisant Experimental Treatment 1 Intervention

All participants receive pitolisant administered orally once daily in the morning upon awakening.

Who Is Running the Clinical Trial?

Harmony Biosciences Management, Inc.

Lead Sponsor

Trials

9

Recruited

2,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40605372/

Pitolisant for EDS in Prader-Willi Syndrome: Study Conclusions: Results of this proof-of-concept study support further evaluation of pitolisant in patients with Prader-Willi syndrome and EDS.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06366464

A Study of Pitolisant in Patients With Prader-Willi Syndrome The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with ...

3. academic.oup.com academic.oup.com/sleep/article/47/Supplement_1/A269/7654669

0631 Phase 2: Pitolisant in Prader-Willi Study Pitolisant reduced EDS in patients with PWS. Pitolisant may also reduce behavioral disturbances and hyperphagia.

4. pwsausa.org pwsausa.org/pitolisant-shows-positive-secondary-outcomes-in-phase-2-study/

Pitolisant Shows Positive Secondary Outcomes in Phase 2 ... In children aged 6 to 12, improvements were noted in various behavioral domains, including irritability and social withdrawal, painting a ...

5. ir.harmonybiosciences.com ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-presents-new-secondary-outcome-data-phase-2

Release Details - Harmony Biosciences HARMONY BIOSCIENCES PRESENTS NEW SECONDARY OUTCOME DATA FROM PHASE 2 SIGNAL DETECTION STUDY IN PATIENTS WITH PRADER-WILLI SYNDROME. October 5 ...