Clinical Trials

Advancing Innovative PWS Treatment Options

Help Advance PWS Care

Clinical trials help scientists develop medications to treat Prader Willi Syndrome. Without research patients, researchers are not able to collect the proper data and samples to determine if therapies are effective or not.

When your child participates in a clinical trial, they are contributing to the advancement of treatment options for individuals living with PWS. Participants are typically compensated and the research is done in a safe, ethical manner.

Rigorous protocols, including informed consent, independent ethics committee reviews, and ongoing monitoring, are in place to ensure the well-being and rights of participants.

Mother and Son of PWS

Find a Trial Near You

Click on the pins for information about trials that are currently recruiting.
We will continue to add clinical trial location sites as they become available.

Clinical Trials 101

Boy with Prader-Willi Syndrome
What is a Clinical Trial?

A clinical trial is a research study assessing the safety, effectiveness, and benefits of new treatments or interventions. Designed with strict protocols involving human participants, these trials compare new approaches to existing ones. They’re essential for advancing medical knowledge and enhancing patient care.

How do Clinical Trials Work?

Participants, selected by criteria, are split into treatment and control groups. The treatment group receives the experiment, while the control might get a placebo or standard care. They’re monitored for safety and outcomes, with committees overseeing progress. After the trial, data is reviewed to gauge the intervention’s safety and efficacy.

PWS Clinical Trial

Clinical Trial Timeline

Phase 1: Safety & Dosing Information

In this initial phase, the treatment’s safety, dosage, and potential side effects are assessed with a small group of healthy volunteers or affected individuals. Researchers monitor participants closely.

Phase 2: Efficacy Studies

In Phase 2, the treatment is tested on a larger group with the specific condition, like Prader-Willi Syndrome. The focus is on evaluating its effectiveness and monitoring for side effects or adverse reactions.

Phase 3: Comparative Effectiveness Study

In this phase, the treatment is compared to current standards or placebos. The goal is to further evaluate its effectiveness, safety, and benefits against other options. Researchers maintain close monitoring and data collection.

Phase 4: Post-Market Monitoring

In Phase 5, the treatment is implemented in clinical practice. Long-term studies assess its real-world effectiveness and longevity beyond trials. These studies offer insights into the treatment’s impact on patient outcomes and quality of life.

Phase 5: Implementation and Follow-up Studies

Phase 5 involves the implementation and dissemination of the treatment in clinical practice. Long-term follow-up studies track patients’ progress and outcomes over an extended period to assess the treatment’s real-world effectiveness, durability, and benefits beyond the clinical trial setting. These studies provide valuable insights into the treatment’s performance and its impact on patients’ lives.

Are Clinical Trials Safe?

Safety is a top priority in clinical trials. Rigorous measures are in place to ensure participant well-being and minimize risks. Before a clinical trial begins, it undergoes a comprehensive review by independent ethics committees or institutional review boards to evaluate its scientific validity and ethical considerations. Additionally, regulatory authorities provide oversight to ensure trials adhere to strict safety guidelines. Throughout the trial, participants are closely monitored by a team of healthcare professionals who are experienced in managing potential risks. Protocols are in place to promptly address any adverse events or side effects that may arise. Moreover, participants have the right to withdraw from the trial at any time if they have concerns about their safety or well-being. The aim of clinical trials is to advance medical knowledge while maintaining the utmost safety for participants.

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Harmony Biosciences Initiates TEMPO PWS Study

Harmony Biosciences Initiates TEMPO PWS Study

Harmony Biosciences Holdings, Inc. is seeking participants for its TEMPO study, a global Phase 3 trial investigating pitolisant as a potential treatment for excessive daytime sleepiness (EDS) in individuals aged six years and older with Prader-Willi syndrome (PWS). Pitolisant is a medication that could help manage sleepiness and behavioral issues in people with PWS. There […]

New PWS Clinical Study: Free Informational Webinar with Harmony Biosciences

New PWS Clinical Study: Free Informational Webinar with Harmony Biosciences

Join PWSA | USA and the team from Harmony Biosciences on Tuesday, March 12th at 8:00 p.m. EST / 5:00 p.m. PST to learn more about the upcoming Phase 3 registrational TEMPO study, a randomized, double-blind, placebo-controlled, multicenter, global clinical study that will further assess the safety and efficacy of pitolisant in patients with PWS, […]

Pitolisant Receives Orphan Drug Designation

Pitolisant Receives Orphan Drug Designation

Pitolisant from Harmony Biosciences Receives Orphan Drug Status from FDA for Treatment of PWS The FDA has granted Orphan Drug status to pitolisant, the trial drug from Harmony Biosciences to treat excessive daytime sleepiness (EDA) and behavioral disturbances. The designation shows that the FDA considers pitolisant to be a promising treatment for people with PWS.  […]

Learn More

Click the logos below to learn more about each pharmaceutical company’s PWS clinical trial.

Drug: ARD-101
Phase: 2

Drug: ACP-101 (Carbetocin)
Phase: 3

Drug: RGH-706
Phase: 2

Drug: Pitolisant
Phase: 3

Drug: NNZ-2591
Phase: 2

Drug: DCCR
Phase: 3 (Complete)