Research Blog

PWS Clinical Trials Alert

 

FPWR maintains the latest clinical trial information so that you can stay abreast of trial opportunities. Some trials require in-person visits while others can be completed remotely.  Some trials are testing new drugs while others are intervention (non-drug) or natural history studies. Details of recruiting studies can be found below.

Pitolisant for Excessive Daytime Sleepiness in PWS [2020 CONFERENCE VIDEO]

In this 30‑minute video, Albena Patroneva from Harmony Biosciences explains what pitolisant is and how it works, and describes Harmony's Phase 2 clinical trial of pitolisant in patients with PWS. The session includes Q&A from participants in the 2020 FPWR Virtual Conference.

Next Steps In PWS Advocacy

For more than a decade, FPWR has partnered with families and other PWS groups (PWSA|USA, IPWSO) to advocate for PWS treatments. We've worked diligently to build resources that address the FDA’s call for patient experience data, demonstrating high disease burden and the need for effective therapies addressing hyperphagia...

FPWR and PWSA|USA Request Regulatory Flexibility & Review of NDA for Intranasal Carbetocin

In a continuation of our joint advocacy efforts supporting the full review of potential drugs for PWS, FPWR and PWSA | USA have submitted a petition letter to the FDA requesting that they apply regulatory flexibility and to review a New Drug Application for intranasal carbetocin, a potential treatment for PWS. Levo Therapeutics recently completed a Phase 3 clinical trial (CARE-PWS) evaluating the safety and efficacy of intranasal carbetocin (

FPWR Announces First Round of 2021 Grant Awards for PWS Research [VIDEO]

The Foundation for Prader-Willi Research announces our first round of Research Awards in 2021 totaling $359,100. FPWR is dedicated to supporting research that advances the understanding and treatment of Prader-Willi syndrome (PWS) and to that end, has awarded over $15,00,000 in research grants since 2003.

FPWR and PWSA | USA Announce Upcoming Meeting with FDA

With your support, FPWR and PWSA | USA have been granted a meeting with the FDA, which will take place via teleconference later this month. This small, non-public meeting will allow FPWR and PWSA | USA to connect first-hand with the FDA staff and share important information that should be considered during the review of new drugs for PWS...

Hyperphagia Leads to Increased Caregiver Burden Across the Lifespan

Hyperphagia is a life-long concern for individuals with PWS and their caregivers. In a recently published study conducted by FPWR and the PWS Clinical Trials Consortium, we found caregiver burden increases as the person with PWS gets older, and as hyperphagia increases. While the findings from this study may not be surprising to PWS families and caregivers, they will be important in our advocacy efforts with pharmaceutical companies and regulatory agencies, like the FDA.

Optimal Nutrition for People With PWS [2020 CONFERENCE VIDEO]

In this 60‑minute workshop, Melanie Silverman explains optimal nutrition for children and adults with Prader‑Willi Syndrome. She includes online resources and gives tips on how to handle feeding times and varying diets. The session includes Q&A from participants in the 2020 FPWR Virtual Conference. 

FPWR Launches First of Kind PWS Genome Project

FPWR is pleased to announce the launch of an exciting new research study: the PWS Genome Project.

This study will provide new insight into how variations across the entire genome influence the range and severity of symptoms in people with Prader-Willi syndrome. The goals of this Prader-Willi syndrome research study are to identify genetic variants that influence the risk and severity of symptoms, and in the long term, understand how DNA variants impact response to different treatments for PWS.

What Does Effective PWS Treatment Advocacy Look Like?

Last fall, the PWS community was excited to see two drugs complete Phase III clinical trials: both DCCR and Carbetocin hold promise in addressing hyperphagia, a top priority for PWS families. Both studies had positive results, but neither reached statistical significance for their primary endpoint: change in hyperphagia...