New drug shows promise for rare genetic disorder in kids
NCT ID NCT07593391
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 study is testing an oral drug called NNZ-2591 in 180 children aged 3 to 12 with Phelan-McDermid syndrome, a rare genetic condition that causes developmental delays and other challenges. The main goal is to see if the drug is safe and tolerable over the long term. Researchers will also check if it helps improve symptoms using a special assessment scale.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
NNZ-2591 (an oral drug taken twice daily)
What this could lead to
If successful, this could provide a long-term treatment option to help manage symptoms and improve daily functioning in children with Phelan-McDermid syndrome.
What could go wrong
This is an open-label extension study, meaning everyone knows they are getting the drug, which can bias results. The trial focuses on safety and may not prove the drug works better than a placebo.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Neuren PMS-302 Site#111
RECRUITINGSan Rafael, California, 94903, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••