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.
Age range
3 Years – 12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants
Timeframe: Baseline through Safety Follow-Up (Month 12)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
Timeframe: Baseline through Safety Follow-Up (Month 12)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
Timeframe: Baseline through Safety Follow-Up (Month 12)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
Timeframe: Baseline through Safety Follow-Up (Month 12)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
Timeframe: Baseline through Safety Follow-Up (Month 12)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
Timeframe: Baseline through Safety Follow-Up (Month 12)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant clinical laboratory parameters events across participants.
Timeframe: Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant physical examination findings across participants.
Timeframe: Baseline through Month 12