NNZ-2591 for Phelan-McDermid Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called NNZ-2591 for children with Phelan-McDermid Syndrome, a condition that can cause developmental delays and other challenges. The goal is to assess the treatment's safety and effectiveness over a long period. The trial seeks children aged 3 to 12 who have completed a previous related study and are not experiencing regression or loss of skills. Participants should not have had more than one seizure in the past year or been on unstable medication regimens. As a Phase 3 trial, this is the final step before FDA approval, offering a chance to contribute to a potentially groundbreaking treatment.
Is there any evidence suggesting that NNZ-2591 is likely to be safe for humans?
Research has shown that NNZ-2591 is generally safe for children and teenagers with Phelan-McDermid Syndrome. Studies indicate that most individuals tolerate the treatment well. Reported side effects were mostly mild, including headaches and fatigue. Both doctors and caregivers observed improvements in key symptoms during treatment. This suggests that NNZ-2591 is not only safe but may also effectively help manage the condition.12345
Why do researchers think this study treatment might be promising for Phelan-McDermid Syndrome?
NNZ-2591 is unique because it's a novel treatment targeting Phelan-McDermid Syndrome at a molecular level. Unlike existing options that mainly address symptoms, NNZ-2591 works by modulating pathways that are directly involved in the condition's underlying mechanisms. Researchers are particularly excited about this treatment because it holds the potential to not just alleviate symptoms, but also to address the root cause of the disorder, offering hope for more substantial and long-lasting improvements in patient outcomes.
What evidence suggests that NNZ-2591 might be an effective treatment for Phelan-McDermid Syndrome?
Research has shown that NNZ-2591, the investigational treatment in this trial, may help treat Phelan-McDermid Syndrome. Previous studies found noticeable improvements in movement, communication, social skills, self-care, and thinking abilities, with participants experiencing significant progress in 10 out of 14 areas measured. The treatment is generally well tolerated, with most side effects being mild to moderate. These results suggest that NNZ-2591 could be a helpful option for managing symptoms of Phelan-McDermid Syndrome.12346
Are You a Good Fit for This Trial?
This trial is for children diagnosed with Phelan-McDermid Syndrome. Participants must meet the study's health requirements and cannot join if they have certain medical conditions or other factors that would make participation unsafe.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive orally administered NNZ-2591 during the 52-week Treatment Period
Follow-up
A 2-week safety follow-up period will occur immediately after the completion of the Treatment Period
Open-label extension
Participants continue to receive NNZ-2591 to evaluate long-term safety, tolerability, and efficacy
What Are the Treatments Tested in This Trial?
Interventions
- NNZ-2591
Trial Overview
The study is testing a medication called NNZ-2591 to see how safe, tolerable, and effective it is when used long-term in children with Phelan-McDermid Syndrome. All participants will receive the medication.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
The total duration of this study for each participant will be up to up to 56 weeks.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Neuren Pharmaceuticals Limited
Lead Sponsor
Citations
NNZ-2591 in Children and Adolescents With Phelan ... - PMC
NNZ-2591 was well tolerated; most treatment-emergent adverse events were mild to moderate. Significant improvements from baseline were observed ...
An Open-Label Study of Oral NNZ-2591 in Phelan ...
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.
NNZ-2591 Phase 2 Publication
Both clinicians and caregivers reported improvements in motor skills, communication, social interaction, self-care skills and cognitive and ...
Safety, efficacy, and exposure-response of NNZ-2591, a ...
Participants receiving NNZ-2591 experienced statistically significant improvement from baseline for 10 of 14 efficacy outcomes, including both clinician- and ...
5.
neurologylive.com
neurologylive.com/view/published-phase-2-data-highlight-potential-nnz-2591-phelan-mcdermid-syndromePublished Phase 2 Data Highlight Potential of NNZ-2591 in ...
A phase 2 trial shows NNZ-2591 improves symptoms in children with Phelan-McDermid syndrome, paving the way for a phase 3 study.
Open-Label-Study-of-Oral-NNZ-2591-in- ...
The purpose of the study is to look at the safety, tolerability, and efficacy of NNZ-2591 in the treatment of children with Phelan-McDermid syndrome.
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