An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001) (NCT05025241) | Clinical Trial Compass
CompletedPhase 2
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
United States18 participantsStarted 2022-08-08
Plain-language summary
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.
Who can participate
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.
Inclusion criteria
. Clinical diagnosis of PMS with a documented disease-causing genetic abnormality of SHANK3.
. Males or females aged 3-12 years.
. Body weight of 12 kg or higher at Screening.
. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
. Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
. Each subject must be able to swallow the study medication provided as a liquid solution.
. Caregiver(s) must have sufficient English language skills.
Exclusion criteria
. Body weight \< 12kg at screening
. Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
. Abnormal QTcF interval or prolongation at Screening.
Questions worth asking your doctor
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
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.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety and Tolerability
Timeframe: 13 weeks
2
Pharmacokinetic - Mean AUC24
Timeframe: Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13.
3
Pharmacokinetic - t1/2
Timeframe: Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13.
. Any other clinically significant finding on ECG at the Screening visit.
. Positive for severe acute respiratory syndrome coronavirus 2 (SARSCoV- 2) and previous COVID 19 infection with last 12 months that required hospitalization
. Unstable or changes Psychotropic treatment 2 weeks prior to screening .
. Excluded concomitant treatments.
. Actively undergoing regression or loss of skills.