FAST Australia is committed to making clinical trials accessible to Australian families in a timely matter.

Trials in Australia

Our goals are to

  • Empower a network of professionals who understand Angelman syndrome, our community, and the outcomes our families want,
  • Enable national and state-wide infrastructure to run and manage clinical trials,
  • Challenge the current obstacles that create excess red tape & make multi-site trials affordable,
  • Connect with all Australian families,
  • Address the under diagnosis of Angelman syndrome in Australia
Enabling

Active Global Angelman Syndrome Research Studies

Roche and Genetech are sponsoring a non drug trial to develop biomarkers for future clinical trials. Participants are individuals diagnosed with AS, as well as typically developing children. (United States)

This is a Phase 1/2, open-label, multiple-dose, dose-escalating study to evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102 in pediatric patients with Angelman syndrome. Approximately 20 patients (male and female) ≥ 4 and ≤ 17 years of age with a genetically confirmed diagnosis of full maternal UBE3A gene deletion (within 15q11.2-q13 region) will be enrolled. (United States) Learn More

  • Cerebralspinal Fluid study

The AS CSF Study sponsored by Biogen is designed to help learn more about AS and find potential biomarkers for use in AS clinical trials in the future. Biomarkers are substances, for example proteins, occurring in the body that can change because of a disease or treatments and can be found in your blood, spinal fluid, and tissue. The AS CSF Study aims to find potential biomarkers that can be measured in the cerebrospinal fluid (CSF), which is the fluid surrounding the brain and spinal cord. Measurement of such biomarkers in AS patients may help guide future disease-modifying drug development. Participants in this study will not receive an investigational drug. (United States)

A Study by Roche/Genentech To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome. To learn more about the Trial click here. (Europe and the United States).

Active studies closed to recruitment

This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in individuals with AS and provide additional OV101 treatment to those individuals who completed Study OV101-15-001 (NCT02996305). Individuals with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria. (Globally including sites in Melbourne and Brisbane) Learn More

Disruptive Nutrition is sponsoring a clinical research study to evaluate the safety and tolerability of a novel nutritional formulation for children with Angelman syndrome. The nutritional formulation can be used with standard, ketogenic, and low glycemic index diets. (United States)