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Oral NNZ-2591 for Phelan-McDermid Syndrome (PMS-001 Trial)

Phase 2
Waitlist Available
Research Sponsored by Neuren Pharmaceuticals Limited
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 13 weeks
Awards & highlights
No Placebo-Only Group

PMS-001 Trial Summary

This trial is testing the safety and effectiveness of a new drug for children and teens with Phelan-McDermid Syndrome.

Who is the study for?
This trial is for children aged 3-12 with Phelan-McDermid Syndrome, weighing at least 12 kg, and not currently losing developmental skills. They must have a SHANK3 genetic abnormality and be able to swallow liquid medication. Exclusions include unstable seizures, certain health conditions like heart or kidney problems, recent severe COVID-19 requiring hospitalization, and changes in psychotropic treatment within the last two weeks.Check my eligibility
What is being tested?
The study tests NNZ-2591's safety, how well it's tolerated by the body (tolerability), its movement through the body (pharmacokinetics), and effectiveness in treating symptoms of Phelan-McDermid Syndrome in children and adolescents.See study design
What are the potential side effects?
While specific side effects are not listed here, common concerns may include reactions related to drug tolerance such as digestive issues, allergic reactions (note allergy exclusion for strawberry), or impacts on mood or behavior given the population being studied.

PMS-001 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~13 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 13 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Pharmacokinetic - Measurement of AUC
Pharmacokinetic - Measurement of Cmax
Pharmacokinetic - Measurement of t1/2
+2 more
Secondary outcome measures
Exploratory efficacy measurement

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

PMS-001 Trial Design

1Treatment groups
Experimental Treatment
Group I: NNZ-2591Experimental Treatment1 Intervention
NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
NNZ-2591
2020
Completed Phase 1
~30

Find a Location

Who is running the clinical trial?

Neuren Pharmaceuticals LimitedLead Sponsor
12 Previous Clinical Trials
926 Total Patients Enrolled
James ShawStudy DirectorNeuren Pharmaceuticals
4 Previous Clinical Trials
88 Total Patients Enrolled

Media Library

NNZ-2591 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05025241 — Phase 2
Phelan-McDermid Syndrome Research Study Groups: NNZ-2591
Phelan-McDermid Syndrome Clinical Trial 2023: NNZ-2591 Highlights & Side Effects. Trial Name: NCT05025241 — Phase 2
NNZ-2591 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05025241 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What potential risks are associated with NNZ-2591 treatment?

"Due to the lack of clinical data regarding efficacy, NNZ-2591 is estimated to be a safety score 2 on our 1–3 scale."

Answered by AI

Does this research endeavor have any age restrictions?

"The age-range criteria for this trial stipulate that participants must be between the ages of 3 and 12. There are 376 studies available for minors, while those older than 65 have access to 983 different clinical trials."

Answered by AI

To what extent is this medical trial involving participants?

"To partake in this medical trial, 20 volunteers must meet the eligibility requirements. These enrollees can come from a wide variety of sites such as Boston Children's Hospital and Rush University Medical Center."

Answered by AI

Is this an original or previously tested experiment?

"NNZ-2591 has been a topic of scientific exploration since 2022, when it was initially studied by Neuren Pharmaceuticals Limited. Following the completion of these initial trials in 2022, NNZ-2591 earned its Phase 2 drug approval and is now being trialed at 11 different locations across two nations."

Answered by AI

Could I potentially join in this experiment?

"This investigation is in search of 20 minors, aged between 3 and 12 years old, who have chromosomal conditions. The principal requirements for enrolment are: a verified diagnosis of PMS due to SHANK3 genetic abnormality; body weight exceeding 12 kg on the day of screening visit; no regression or diminishment of skills within last three months before screening visit; English-speaking caregivers; CGI-S score equal or greater than 4 at Screening Visit; ability to swallow study medication as liquid solution."

Answered by AI

Are there any open enrollment opportunities for this medical trial?

"That is correct. According to clinicaltrials.gov, this medical research opened for recruitment on August 8th 2022 and was last modified on November 15th of the same year. It is currently seeking 20 participants from 5 different sites."

Answered by AI

To what extent has this research been implemented across healthcare facilities?

"The trial has 5 sites in operation, including Boston, Chicago and Houston. To reduce travel time and costs, it is advisable to choose a clinic close-by if you decide to join the study."

Answered by AI

Is there any precedent of research involving NNZ-2591?

"Presently, 3 clinical trials related to NNZ-2591 are in operation. None of them have reached the Phase 3 stage yet. The bulk of these studies take place at a single location in Greenwood, South carolina; however, there are 13 other medical centres running similar tests for this drug."

Answered by AI

Who else is applying?

What state do they live in?
California
Virginia
Ohio
Other
How old are they?
< 18
18 - 65
What site did they apply to?
Rush University Medical Center
Texas Children's Hospital
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria

How responsive is this trial?

Typically responds via
Email
~8 spots leftby Feb 2025