New drug shows promise for rare genetic disorder in kids
NCT ID NCT07593391
First seen Jun 26, 2026 · Last updated Aug 13, 2026 · Updated 5 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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Neuren PMS-302 Site#104
RECRUITINGLexington, Massachusetts, 02421, United States
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Neuren PMS-302 Site#108
RECRUITINGCincinnati, Ohio, 02421, United States
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Neuren PMS-302 Site#109
RECRUITINGChevy Chase, Maryland, 20815, United States
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Neuren PMS-302 Site#111
RECRUITINGSan Rafael, California, 94903, United States
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Neuren PMS-302 Site#115
RECRUITINGHouston, Texas, 77030, United States
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Neuren PMS-302 Site#122
RECRUITINGGlendale, California, 91203, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can early parent coaching help infants with rare genetic disorders thrive?
- Gene therapy for rare genetic syndrome moves to Long-Term safety check
- Gene therapy injection into the brain aims to treat rare genetic disorder
- Hope for rare genetic disorder: new drug trial targets core symptoms
- Light-Based brain cap could replace radiation scans for kids