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
neu-2591-pws-001@
Uncommon Cures
Chevy Chase, MD (near Washington DC)
Recruitment coordinator Henry Nickson: PWStrialreferral@precisionformedicine.com
Visit ClinicalTrials.Gov for the most current list of sites.
Questions?
You can learn more about this study at: www.pws-trial.com
For questions on eligibility and locations, please email: PWStrialreferral@precisionformedicine.com
