Clinical trials

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

Active global Angelman syndrome research studies

FREESIAS Endpoint Study

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. This trial will recruit two different age cohorts; 1-4yrs and 5-12yrs. To learn more about the Trial click here. (Europe and the United States).

Should you relocate to access a clinical trial?

There are several important factors which should be considered prior to resorting to relocating for clinical trial recruitment. At this point in history, one of the most significant considerations is COVID19. Participants will be put into a high travel burden; Participants are at a higher risk of contracting COVID-19 due to increased travelling requirements.

The next important consideration is eligibility. Trials that are listed on will list inclusion and exclusion criteria that is set by the sponsor conducting the trial and accepted by the regulatory authority in that country. These criteria are only able to be changed by the sponsor and would require   submitting an amended trial protocol to the relevant authority.  Only the sponsor in charge of the specific clinical trial can determine whether or not an individual meets the inclusion criteria and none of the exclusion criteria in order to qualify for participation in their clinical study.  If an individual meets all of the inclusion criteria and none of the exclusion criteria, it is typically the Primary Investigator of the study who chooses an eligible patient. The distance a patient would need to travel in order to participate is likely an important factor in the decision making process. Future trials that are not listed on have not yet determined recruitment criteria.

Migration. Unless you are a resident or immediate family member most countries have a process to apply for migration, this process needs to be done prior to entry into the country of migration. The United States and many other countries, have various types and requirements when it comes to visas for international visitors. The United States, for example, can issue visitor visas for a period of 90 days to countries who participate in the Electronic System for Travel Authorization. (ESTA) program.

Whilst there are no costs for participation in a clinical trial, access to medical care in some countries can be extremely expensive without insurance and this should be taken into consideration.

It is important to remember that one of the primary goals for FAST Australia is to make clinical trial participation available to Australian families, we will continue to strive towards this as more and more trials come to fruition.

Active studies closed to recruitment

Phase 3 Neptune (Ovid)

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

FANS Nutritional formulation in Angelman syndrome

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)