๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-08-10

An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome (NCT07593391)

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

๐ŸŸข Recruiting Phase3 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: Neuren Pharmaceuticals Limited

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 in pediatric participants with Phelan- McDermid Syndrome.

Who can join: Review the eligibility criteria below.

Location: Neuren PMS-302 Site#122 โ€” Glendale, California

Age range: 3 Years to 12 Years

๐Ÿงช Interventions in This Study

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (6 Sites)

  • Neuren PMS-302 Site#122 โ€” Glendale, California
  • Neuren PMS-302 Site#111 โ€” San Rafael, California
  • Neuren PMS-302 Site#109 โ€” Chevy Chase, Maryland
  • Neuren PMS-302 Site#104 โ€” Lexington, Massachusetts
  • Neuren PMS-302 Site#108 โ€” Cincinnati, Ohio
  • Neuren PMS-302 Site#115 โ€” Houston, Texas

๐Ÿ“ž Contact the Study Coordinator

Phone: (231) 203-8050

Email: medicalinformation@neurenpharma.com

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

๐Ÿ’ฐ Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Contact the study coordinator using the phone/email above.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—