Important information regarding this exciting milestone:
- Rare Disease Research (RDR), located in Atlanta, GA, is now welcoming children with PWS and their families to their clinicfor screening into this trial.
- The duration of active treatment in this study is 13 weeks. In a preclinical study in animals, physiological and behavioral symptoms were normalized within six weeks of dosing.
- Enrollment will begin in children diagnosed with PWS who are in the older age group (ages 8 through 12).
- After safety and tolerability data in the older group is independently reviewed, it will be announced when children in the younger group (ages 4 through 7) can start to be screened.
- As this is an open-label study, all children who participate on this trial will receive study drug.
- All travel and lodging for in-clinic visits (5 in total) can be reimbursed.
- If the study drug (NNZ-2591) development moves into Phase 3, all children who participate in this Phase II study may be eligible for an Open-label Extension study that would be opened in parallel to the NNZ-2591 Phase III trial in PWS.
- Study goals:
- The overall goal of this first NNZ-2591 clinical trial in children with PWS is to assess safety and inform the design of subsequent registration trials.
- The primary outcome measures in the study are safety and tolerability as well as measuring how the drug is used and distributed in the body.
- The secondary goal is to assess the potential benefit of NNZ-2591. The study looks at a range of outcomes that are functionally important to children with PWS: behavioral issues, anxiousness, social interaction, language, cognition/learning, hyperphagia/food behaviors, sleep, and activities of daily living.
For any questions in relation to study participation at Rare Disease Research, please reach out directly to the site at neu-2591-pws-001@rarediseaseresearch.com (please note that his email address is actively monitored for incoming emails in need of response). For any general questions, please reach out to Dorothea Lantz (dlantz@pwsausa.org).
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Jennifer Bolander has been serving as a Special Education Specialist for PWSA (USA) since October of 2015. She is a graduate of John Carroll University and lives in Ohio with her husband Brad and daughters Kate (17), and Sophia (13) who was born with PWS.
Perry A. Zirkel has written more than 1,500 publications on various aspects of school law, with an emphasis on legal issues in special education. He writes a regular column for NAESP’s Principal magazine and NASP’s Communiqué newsletter, and he did so previously for Phi Delta Kappan and Teaching Exceptional Children.
Evan has worked with the Prader-Willi Syndrome Association (USA) since 2007 primarily as a Crisis Intervention and Family Support Counselor. Evans works with parents and schools to foster strong collaborative relationships and appropriate educational environments for students with PWS.
Dr. Amy McTighe is the PWS Program Manager and Inpatient Teacher at the Center for Prader-Willi Syndrome at the Children’s Institute of Pittsburgh. She graduated from Duquesne University receiving her Bachelor’s and Master’s degree in Education with a focus on elementary education, special education, and language arts.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Program (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Children’s Hospital in Denver supporting families and school districts around the United States with their child’s Individual Educational Plan.
Founded in 2001, SDLC is a non-profit legal services organization dedicated to protecting and advancing the legal rights of people with disabilities throughout the South. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act. Recently in November 2014, Jim retired.