Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug shows promise for rare genetic disorder in kids

NCT ID NCT07593391

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Sep 21, 2026 · Updated 6 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 180 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Nov 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 12 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent for the antecedent study. 2. Participant must have completed all applicable study visits for the antecedent study in which they participated. 3. Body weight ≥ 10 kg at Screening/Baseline. 4. Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits. 5. Not actively undergoing regression or loss of skills. Exclusion Criteria: 1. Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol. 2. Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications). 3. Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regimen that have not been stable for at least 4 weeks before Screening. If the treatment was discontinued, the discontinuation must have occurred no fewer than 2 weeks before the start of Screening. 4. Any intercurrent seizures in the past 6 months and /or more than 1 seizure in the past 12 months. •A single febrile seizure in the 6 months prior to screening is allowable if no rescue medication was required. 5. Abnormal liver function laboratory results during the Screening period, as defined by the protocol 6. Abnormal QT interval on Screening ECG as defined by the protocol.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Phelan-mcdermid syndrome are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    9 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Neuren PMS-302 Site #116

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • Neuren PMS-302 Site#101

    RECRUITING

    New York, New York, 10029, United States

  • Neuren PMS-302 Site#104

    RECRUITING

    Lexington, Massachusetts, 02421, United States

  • Neuren PMS-302 Site#106

    RECRUITING

    Brookline, Massachusetts, 02445, United States

  • Neuren PMS-302 Site#108

    RECRUITING

    Cincinnati, Ohio, 02421, United States

  • Neuren PMS-302 Site#109

    RECRUITING

    Chevy Chase, Maryland, 20815, United States

  • Neuren PMS-302 Site#111

    RECRUITING

    San Rafael, California, 94903, United States

  • Neuren PMS-302 Site#115

    RECRUITING

    Houston, Texas, 77030, United States

  • Neuren PMS-302 Site#122

    RECRUITING

    Glendale, California, 91203, United States

More trials for these conditions

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