PWSA Blog

Wednesday January 26 2024 800 PM EST 3, Prader-Willi Syndrome Association | USA

Neuren Pharmaceuticals Opens Second Trial Site for Phase II PWS Study

Neuren Pharmaceuticals is pleased to announce their second site participating in their Phase II, Open Label, PWS Study (Neu-2591-PWS-001) is now open for screening!

Important information regarding this exciting milestone:

  • Two sites are now open to enrollment! Rare Disease Research (RDR), located in Atlanta, GA, and Uncommon Cures, located in Chevy Chase, MD (8 miles outside of Washington, D.C.) are now welcoming children with PWS and their families to their clinic for screening into this trial. 
  • A third site in Philadelphia, PA is expected to be open for screening before the end of this month.
  • 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 currently continues 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 (which will commence following the treatment of 3 patients in the older age group), 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 the study drug (NNZ-2591). 
  • All travel and lodging for in-clinic visits (5 in total) can be fully reimbursed for patients and their families.
  • If 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 3 trial in PWS. 

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