Hope for rare genetic disorder: new drug trial targets core symptoms

NCT ID NCT07281079

Recruiting now Disease control Sponsor: Neuren Pharmaceuticals Limited Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Aug 13, 2026 · Updated 5 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Phelan-McDermid syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHELAN-MCDERMID SYNDROME

Contacts and locations

Locations

Neuren PMS-301 Site #112
RECRUITING

Palo Alto, California, 94305, United States
Neuren PMS-301 Site #116
RECRUITING

Birmingham, Alabama, 35233, United States
Neuren PMS-301 Site #117
RECRUITING

Ann Arbor, Michigan, 48109, United States
Neuren PMS-301 Site#101
RECRUITING

New York, New York, 10029, United States
Neuren PMS-301 Site#102
RECRUITING

Chicago, Illinois, 60612, United States
Neuren PMS-301 Site#103
RECRUITING

San Diego, California, 92123, United States
Neuren PMS-301 Site#104
RECRUITING

Lexington, Massachusetts, 02421, United States
Neuren PMS-301 Site#106
RECRUITING

Brookline, Massachusetts, 02445, United States
Neuren PMS-301 Site#108
RECRUITING

Cincinnati, Ohio, 45229, United States
Neuren PMS-301 Site#109
RECRUITING

Chevy Chase, Maryland, 20815, United States
Neuren PMS-301 Site#111
RECRUITING

San Rafael, California, 94903, United States
Neuren PMS-301 Site#115
RECRUITING

Houston, Texas, 77030, United States
Neuren PMS-301 Site#122
RECRUITING

Glendale, California, 91203, United States
Neuren PMS-301 Site#201
RECRUITING

Toronto, Ontario, M4G 1R8, Canada

More trials for these conditions

Other studies related to the condition(s) this trial covers.