NNZ-2591 as a Potential Therapeutic for Children with PWS

Study Purpose

This open-label study will study the safety, tolerability, pharmacokinetics, and efficacy of NNZ-2591 in children with PWS ages 4 to 12 years. Study participants will receive NNZ-2591 oral solution ( a ready-to-drink strawberry-flavored liquid) twice daily for 13 weeks. Every participant will receive NNZ-2591. 

Participation involves 5 trial visits to the study site over the course of the study. All travel and lodging expenses will be reimbursed. 

Enrollment will begin with children ages 8-12. After safety and tolerability data has been reviewed, children ages 4-7 will be able to enroll. 

Recruitment Criteria

Study Type: Interventional
Eligible Ages: 4 - 12 years (currently enrolling 8-12)
Other Criteria: 

To be in the study, participants must meet the following basic criteria:

  • Are between the ages of 4 and 12 years
  • Have a diagnosis of PWS
  • Have a body weight between 12 kg and 100kg at baseline
  • Must currently be on growth hormone
  • Person with PWS and caretaker must reside in the US

Medical history and other criteria will also be reviewed to determine eligibility. See the trial listing at clinicaltrials.gov for a full list of inclusion and exclusion criteria. 

Trial Details

ClinicalTrials.Gov Id: NCT05879614
Phase: 2
Duration: 13 weeks
Lead Sponsor: Neuren
Countries: United States, Australia

This study is available at the following sites:  

Rare Disease Research (RDR)
Atlanta, GA

Uncommon Cures
Chevy Chase, MD (near Washington DC)
Recruitment coordinator Henry Nickson:

Visit ClinicalTrials.Gov for the most current list of sites.                                                                                     


You can learn more about this study at: www.pws-trial.com
For questions on eligibility and locations, please email
:   PWStrialreferral@precisionformedicine.com




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