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NCT06366464RECRUITINGIRB Ready

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Sponsored by Harmony Biosciences Management, Inc.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 134 participants interested
1% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living 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 โ‰ฅ6 years of age with Prader-Willi syndrome.

Who Can Participate

Inclusion Criteria

Genetically confirmed diagnosis of PWS
Excessive daytime sleepiness
Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.
In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.

Exclusion Criteria

Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled
Has a diagnosis of hypersomnia due to another sleep/medical disorder
Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

134 participants

โšง

Gender

ALL

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Sponsor

Harmony Biosciences Management, Inc.

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
RECRUITING

Sacramento

California

Location available
View Sacramento location page
RECRUITING

San Diego

California

Location available
View San Diego location page
RECRUITING

San Ramon

California

Location available
View San Ramon location page
RECRUITING

Aurora

Colorado

Location available
View Aurora location page
RECRUITING

Wilmington

Delaware

Location available
View Wilmington location page
WITHDRAWN

Atlanta

Georgia

Location available
View Atlanta location page
RECRUITING

Atlanta

Georgia

Location available
View Atlanta location page
WITHDRAWN

Atlanta

Georgia

Location available
View Atlanta location page

๐Ÿ“ And 13 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Prader-Willi Syndrome Treatment in Los Angeles?

Join others in California exploring innovative treatment options through clinical research

Prader-Willi Syndrome Treatment Options in Los Angeles, California

If you're searching for Prader-Willi Syndrome treatment in Los Angeles, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Los Angeles, Sacramento, San Diego and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Prader-Willi Syndrome. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 134 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Prader-Willi Syndrome?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Prader-Willi Syndrome

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Prader-Willi Syndrome Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06366464. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.