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 firstname.lastname@example.org (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 (email@example.com).