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Oral NNZ-2591 for Phelan-McDermid Syndrome (PMS-001 Trial)
PMS-001 Trial Summary
This trial is testing the safety and effectiveness of a new drug for children and teens with Phelan-McDermid Syndrome.
PMS-001 Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.PMS-001 Trial Design
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Who is running the clinical trial?
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- You are currently experiencing a decline or loss of abilities.You have frequent or unpredictable seizures that are difficult to control.You have had cancer in the past or currently have cancer.You are allergic to strawberries.You have a history of stroke or brain injury.You have a thyroid condition that needs treatment or uncontrolled diabetes requiring insulin.You have experienced or are currently experiencing symptoms that make you appear stiff or unresponsive.You currently have a serious or ongoing depression, including bipolar depression.You have a significant problem with your eyesight or hearing that can't be fixed with glasses or hearing aids.
- Group 1: NNZ-2591
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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."
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."
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."
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."
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."
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."
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."
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."
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