Hope for rare genetic disorder: new drug trial targets core symptoms
NCT ID NCT07281079
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an oral drug called NNZ-2591 in 160 children aged 3 to 12 with Phelan-McDermid syndrome, a rare genetic condition. The drug is compared to a placebo to see if it improves communication and daily function. The trial is double-blind, meaning neither the families nor the doctors know who gets the real drug.
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 it works, this could provide a treatment to improve communication and daily living skills in children with Phelan-McDermid syndrome.
What could go wrong
This is a Phase 3 trial, but it is still experimental. The drug may not work better than placebo, and side effects are possible. Results may not apply to all patients.
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-301 Site#101
RECRUITINGNew York, New York, 10029, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#102
RECRUITINGChicago, Illinois, 60612, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#103
RECRUITINGSan Diego, California, 92123, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#104
RECRUITINGLexington, Massachusetts, 02421, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#106
RECRUITINGBrookline, Massachusetts, 02445, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#108
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#109
RECRUITINGChevy Chase, Maryland, 20815, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#111
RECRUITINGSan Rafael, California, 94903, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Neuren PMS-301 Site#122
RECRUITINGGlendale, California, 91203, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••