The Foundation for Prader-Willi Research and Zafgen Partner for PATH for PWS Natural History Study

The Foundation for Prader-Willi Research (FPWR) and Zafgen, Inc. have partnered to execute a 4-year study that will advance the understanding of the natural history and medical events in people with Prader-Willi syndrome (PWS). The PATH for PWS study (Paving the way for Advances in Treatments & Health for PWS) is a non-interventional, observational study of serious medical events in PWS, intended to inform the development and clinical trial design for potential new treatments for PWS.

“It is critical that we continue to build on the body of knowledge to improve care and explore new treatment options for PWS,” said Theresa Strong, Ph.D., Director of Research Programs, FPWR, and the lead study investigator. “The PATH for PWS study will allow us to collect and analyze important health information that will inform PWS clinical trial design, new and better treatment options, and additional resources for families and physicians.”

The PATH for PWS Study brings together multiple stakeholders including Zafgen, patient groups (FPWR, and the Prader-Willi Syndrome Association (USA) (PWSA-USA)), as well as key PWS clinicians – Dr. Jennifer Miller, Pediatric Endocrinologist at University of Florida and Dr. Shawn McCandless, Medical Geneticist at Children’s Hospital Colorado. The study includes measures of specific PWS behaviors, which will be monitored over time.

“The Institute of Medicine refers to natural history studies in rare diseases as the pillars of epidemiologic research on rare conditions, and FPWR will continue to support such foundational research,” says John Walter, CEO, FPWR. “We encourage caregivers of adults and children living with PWS to consider participating in the PATH for PWS to contribute to the actionable information available to our community.”

“We are confident that those with PWS will benefit from this study, now and in the future, in many ways including but not limited to patient care, treatment best practices, and clinical trial design and readiness,” says Steve Queior, CEO, PWSA-USA.

Are you interested in participating in the PATH study?

Every six months, caregivers of people with PWS enrolled in the study will be asked to provide an update on any serious medical issues experienced and related medical procedures or prescriptions, as well as information about conditions often associated with PWS such as hyperphagia. Clinic visits will not be required, as the information will be provided through Internet-based surveys and questionnaires. The data will be analyzed to evaluate the incidence of serious medical events, prescription medication use associated with medical events of interest, patterns of hyperphagia-related behaviors, as well as additional areas of importance to the PWS community such as changes in body weight, management strategies for hyperphagia, and characterization of traits and actions common to individuals with PWS. The analyses will also help the Prader-Willi research community identify potential areas for future study.

To be eligible for the study, patients must have a confirmed diagnosis of PWS, be at least 5 years of age, live in the United States, Canada or Australia, and be enrolled or willing to enroll in the Global PWS Registry. The primary caregiver of the enrolled person with PWS must have access to the internet to enter study data and consent to being contacted by registry staff and allowing registry staff to enter specific data on their behalf. Participation in clinical trials and other non-interventional studies will be permitted while enrolled in PATH for PWS.

Enrollment for the four-year study is anticipated to begin in September 2018 through the Global Prader-Willi Syndrome Patient Registry, which is powered by the National Organization for Rare Disorders’ (NORD) IAMRARE™ Registry Program.

Ahead of the PATH for PWS study enrollment opening in September 2018, people with PWS and their caregivers can enroll in the broader Global PWS Patient Registry at

If you would like to receive notifications when the study is open and enrolling participants, you may join our interest list here.

Source: The Foundation for Prader-Willi Research


Categories: Pharmaceuticals and Biotech

Tags: hyperphagia, natural history, Prader-Willi Syndrome, PWS, rare disease, registry

About Foundation for Prader-Willi Research

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Established in 2003, the mission of FPWR is to eliminate the challenges of Prader-Willi syndrome through the advancement of research and therapeutic development.

Foundation for Prader-Willi Research
340 S Lemon Ave #3620
Walnut, CA 91789
United States