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Prader-Willi research

Zafgen and Prader-Willi Foundation agree PATH research

Information and data from Global Prader-Willi Syndrome Patient Registry will advance understanding of medical history and medical events in people with PWS
People living with PWS ages 5 and older and their caregivers are encouraged to participate

Zafgen and the Foundation for Prader-Willi Research (FPWR), a non-profit organisation founded to eliminate the challenges of Prader-Willi syndrome (PWS) through the advancement of research and therapeutic development, have announced the launch of a co-sponsored natural history study to advance understanding of the medical history of and medical events in people with PWS.

PATH for PWS (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 development and clinical trial design for potential new treatments for PWS, including ZGN-1258, Zafgen’s new, second-generation programme for PWS designed to decrease hyperphagia, change the way the body metabolizes fat and reduce fat mass.

Enrolment 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. 

“While we understand more about Prader-Willi syndrome than ever before, it is critical that we continue to build on the body of knowledge to improve care and explore new treatment options,” said Dr Theresa Strong, Director of Research Programs, FPWR, and the lead study investigator. “The PATH for PWS study will allow us to collect and analyse important health information about those with PWS to better understand how the condition presents and how that could inform clinical trial design, new and better treatment options, and even additional resources for families and physicians. We hope caregivers of adults and children living with PWS will consider participating in the PATH for PWS to contribute to the actionable information available to our community.”

Every six months, caregivers of people with PWS enrolled in the study will be asked to provide an update on any medical issues experienced and related medical procedures or prescriptions, and 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. 

“PATH for PWS is a testament to the community-wide commitment to PWS. This isn’t about a single therapy, but about the power of a partnership between PWS research and advocacy organizations, industry and the broader community to transform outcomes for patients and their families through greater knowledge and understanding,” said Jeff Hatfield, Chief Executive Officer, Zafgen.

The data will be analysed to evaluate the incidence of serious medical events, prescription medication use associated with medical events of interest, patterns of hyperphagia-related behaviours, 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 Zafgen, FPWR and others in the Prader-Willi research community identify potential areas for future study.

“Natural history studies are a best practice in rare disease drug development, and we are looking forward to important insights that the PATH for PWS study will provide into medical events experienced by people with PWS,” said Dr Tom Hughes, President and Chief Scientific Officer, Zafgen. “As we continue work on our ZGN-1258 program, the PATH for PWS study will be an invaluable resource to inform its development and potentially other therapeutic development work for those with PWS.”

To be eligible for the study, patients must have a confirmed diagnosis of PWS, be at least five years of age, live in the US, Canada or Australia, and be enrolled or willing to enrol 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.

FPWR is composed of thousands of parents, family members, researchers, and others who are interested in addressing the many issues related to PWS, including childhood obesity, developmental delays, psychiatric disorders, and autism spectrum disorders. The mission of FPWR is to eliminate the challenges of Prader-Willi syndrome through the advancement of research and therapeutic development. FPWR supports cutting edge research studies around the world to advance the understanding of PWS, and collaborates with research institutions, pharmaceutical companies and patient advocacy groups to advance new treatments that will help those with PWS.  To date, FPWR has funded over US$10 million in PWS research.

For more information about PATH for PWS, please click here

Ahead of PATH for PWS study enrolment opening in September 2018, people with PWS and their caregivers can enrol in the broader Global PWS Patient Registry here

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