NNZ-2591 for Phelan-McDermid Syndrome in Children
This study is testing a drug called NNZ-2591 for children with Phelan-McDermid Syndrome. It's an open-label study, meaning everyone knows they are receiving the active drug. The main goal is to see how safe NNZ-2591 is over a long period, looking at any side effects and changes in heart activity (ECG parameters) for up to 12 months. The study plans to enroll 180 children aged 3 to 12 years old who have previously participated in other studies for NNZ-2591. The drug will be given by mouth twice a day.
- Study design
- This is an open-label extension study, meaning all 180 participants will receive the study drug. It is designed to evaluate the long-term safety and tolerability of NNZ-2591.
- What's involved
- You would receive NNZ-2591 orally twice daily for 52 weeks. There will be assessments for eligibility, baseline characteristics, and symptom severity, followed by a 2-week safety follow-up period.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for safety for 2 weeks after the 52-week treatment period, with safety and heart activity (ECG) monitored for up to 12 months.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
At a glance
Conditions
NCT07593391
Where you'd take part
This study runs at 6 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Neuren PMS-302 Site#104
Lexington, Massachusettsstudy coordinator listed
Recruiting
Neuren PMS-302 Site#108
Cincinnati, Ohiostudy coordinator listed
Recruiting
Neuren PMS-302 Site#109
Chevy Chase, Marylandstudy coordinator listed
Recruiting
Neuren PMS-302 Site#111
San Rafael, Californiastudy coordinator listed
Recruiting
Neuren PMS-302 Site#115
Houston, Texasstudy coordinator listed
Recruiting
Neuren PMS-302 Site#122
Glendale, Californiastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participantsBaseline through Safety Follow-Up (Month 12)
Incidence of TEAEs, AESI and SAEs across participants
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.Baseline through Month 12
Change from Baseline in ECG Heart Rate (bpm)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.Baseline through Safety Follow-Up (Month 12)
Change from Baseline PR Interval (ms QRS interval (ms)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.Baseline through Safety Follow-Up (Month 12)
Change from Baseline in QT interval (ms)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.Baseline through Safety Follow-Up (Month 12)
Change from Baseline in QTcB interval (ms)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.Baseline through Safety Follow-Up (Month 12)
Change from Baseline in QTcF interval (ms)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.Baseline through Safety Follow-Up (Month 12)
Change from Baseline in RR interval (ms)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.Baseline through Month 12
Change from Baseline for heart rate (bpm)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.Baseline through Month 12
Change from Baseline for respiration rate (breaths per minute)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.Baseline through Month 12
Change from Baseline for Temperature (Celsius)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.Baseline through Month 12
Change from Baseline for Diastolic Blood Pressure (mm Hg)
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.Baseline through Month 12
Change from Baseline for Systolic Blood Pressure (mm Hg)
- Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant clinical laboratory parameters events across participants.Baseline through Month 12
Incidence of abnormal and clinically significant laboratory parameters
- Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant physical examination findings across participants.Baseline through Month 12
Incidence of abnormal, clinically significant physical examination findings