PATH for PWS, a Prader-Willi Study, to Start Enrolling Patients in September

Zafgen biopharma and the nonprofit Foundation for Prader-Willi Research (FPWR) have launched “PATH for PWS,” a natural history study aimed at advancing the understanding of the medical history and medical events in patients with Prader-Willi syndrome (PWS).

The non-interventional, observational study aims to document serious medical events experienced by Prader-Willi patients to improve the design of future clinical trials for potential new treatments.

These include ZGN-1258, Zafgen’s investigative therapy for Prader-Willi syndrome, designed to change the way the body metabolizes fat, reduce fat mass, and decrease excessive hunger (called hyperphagia).

“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,” Theresa Strong, PhD, director of research programs at FPWR and the study’s lead investigator, said in a press release.

“The PATH for PWS study will allow us to collect and analyze 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,” she said.

The study will gather data not only on the medical issues experienced by patients, but also on all medical procedures, prescriptions, and other health conditions associated with Prader-Willi, such as hyperphagia and changes in body weight.

Patients will not be required to visit their physicians for these assessments, since all the information will be collected online every six months from their primary caregivers.

“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,” Strong said.

Researchers are confident the findings from the study will also help Zafgen, FPWR, and others in the Prader-Willi research community identify new areas for future studies.

“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,” said Tom Hughes, PhD, president and chief scientific officer at 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,” Hughes added.

Enrollment for the four-year study is expected to begin in September 2018 through the Global PWS Registry, which is powered by the National Organization for Rare Disorders’ (NORD) IAMRARE Registry Program.

To be eligible for the study, patients must have a confirmed diagnosis of Prader-Willi syndrome, be at least 5 years old, live in the United States, Canada, or Australia, and be enrolled in the Global PWS Registry.

In addition, the primary caregiver of the patient enrolled must have access to the internet be able to enter all the medical information required via online-based surveys and questionnaires.

Patients and caregivers interested in participating in the study can register now at the Global PWS Registry.