PMS-302 – The Koala OLE Study
Study Title:
A Phase 3 Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Orally Administered NNZ-2591 in Pediatric Participants with Phelan-McDermid Syndrome
About the Koala OLE Study:
The Koala OLE Study is evaluating the long-term effects of the investigational study drug, NNZ-2591, in pediatric participants with Phelan-McDermid Syndrome (PMS).
There is no placebo in the Koala OLE Study, and all participants will receive NNZ-2591 investigational drug treatment as a liquid orally administered twice daily for up to 52 weeks. (Reminder: a placebo is an inactive treatment that looks like NNZ-2591 but does not contain any active ingredient.) Therefore, caregivers, participants, the study doctors and their teams, and the sponsor, Neuren Pharmaceuticals Ltd. (Neuren), all know exactly which treatment is being given. This is called ‘open-label’ treatment.
Who can take part in the Koala OLE Study?
The Koala OLE Study is enrolling pediatric participants with PMS who completed either the Neuren Phase 3 Koala Study NEU-2591-PMS-301 (PMS-301) or the Phase 2 Study NEU-2591-PMS-001 (PMS-001).
- Koala Study (PMS-301) Study Participants: If your child is continuing from this Koala Study, data from the PMS-301 study final visit will be used to check if it is ok for them continue in the Koala OLE Study.
- Phase 2 Study PMS-001 Study Participants: If your child participated in the previous Phase 2 Study PMS-001 they will have tests and assessments done during a 4-6 weeks screening period to see if they meet all of the requirements to participate.
Your child may be 1 of approximately 180 pediatric participants taking part in this study in about 30 locations in the United States, Canada, and possibly several other countries.
How long will the Koala OLE Study last?
Your child would be expected to participate in the Koala OLE Study for at least 1 year. The Koala OLE study will include a screening/baseline period, a 52-week treatment period with an open-label investigational drug NNZ-2591 as a liquid to be administered orally twice daily (in which there will be on-site clinic visits and remote visits) and a 2-week follow-up period (following the last dose of the NNZ-2591).
Your child will be closely monitored by study doctors throughout the Koala OLE Study treatment period.
Taking part in a research study is voluntarily and involves risk. Please refer to the Frequently Asked Questions (FAQ) Tab for more information and other Koala study questions.
You can visit https://clinicaltrials.gov/study/NCT07593391 for more information on the study assessments, entry criteria and site locations. For additional site locations, please contact Neuren’s Medical Information Team: medicalinformation@neurenpharma.com