Clinical Trial Initiation for Drug NNZ-2591 to Treat Angelman Syndrome Commences in Australia
In an exciting development for the medical community, a Phase 2 trial of an investigational oral therapy named NNZ-2591 is currently recruiting pediatric patients in Australia. This trial, aimed at treating Angelman syndrome, is being conducted at three hospitals: one each in Queensland, New South Wales, and Victoria.
NNZ-2591 is a compound with a chemical structure similar to the cyclic glycine proline. Its purpose is to restore the normal communication and signaling between brain cells, which are affected in individuals with Angelman syndrome.
Eligible children, aged between 3 and 17, with a confirmed diagnosis of Angelman syndrome are being enrolled at the Centre for Clinical Trials in Rare Neurodevelopmental Disorders at Children's Health Queensland Hospital in South Brisbane, Queensland. The trial plans to enroll up to 20 children in total.
Following an initial four-week observation and assessment period, all children will be given NNZ-2591 as an oral solution twice daily for 13 weeks, approximately three months. A follow-up assessment will be conducted two weeks after finishing treatment.
The main goal of the trial is to assess NNZ-2591's safety and tolerability, and its pharmacokinetics. Additionally, exploratory measures of treatment efficacy will be evaluated, including improvements or worsening of patients using various Angelman syndrome-specific Clinical Global Impression scales, and caregivers' impressions of change.
Preclinical studies with mice genetically engineered to lack the UBE3A gene, the gene commonly mutated in Angelman syndrome, have shown that treatment with NNZ-2591 improved cognition and motor function, reduced anxiety, and prevented seizures.
Top-line results from the Phase 2 trial are expected in May 2023. Jon Pilcher, CEO of Neuren, the company behind NNZ-2591, expressed excitement about the trial in a press release.
The trial has received regulatory clearance in the U.S., and detailed site information and general contact information are available for the trial. The open-label and dose-escalating study (NCT05011851) aims to aid in the design of a larger and registrational trial for NNZ-2591's approval to treat Angelman syndrome.
The Phase 2 study of the oral therapy NNZ-2591 for Angelman Syndrome by Neuren Pharmaceuticals involves research facilities in the states of New South Wales and Victoria, Australia, but specific institutions are not named in the available sources. The main Neuren Pharmaceuticals office is located in Camberwell, Victoria.
This trial marks a significant step forward in the pursuit of a potential treatment for Angelman syndrome, a rare genetic disorder characterised by severe intellectual disability, speech impairment, and seizures.
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