Phase 2 Parkinson's Disease Trial, Not Yet Recruiting
voxsanity.com.au · Eligibility summary from public government registries · 18 August 2026 · not medical advice
Who may and may not be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who may be able to join
- People aged 30 to 70 years (inclusive) at the time of signing the consent form, either male or female.
- People who have been diagnosed with Parkinson's disease caused by a mutation in the GBA1 gene, confirmed through medical history and meeting recognised international diagnostic criteria, with at least one confirmed harmful GBA1 gene mutation.
- People whose glucocerebrosidase (GCase) enzyme activity — a specific enzyme measured in the blood — has been found to be below the normal range in past or screening tests.
- People whose Parkinson's disease is at a moderate-to-severe stage (Hoehn-Yahr stage 3 to 4) when in the "OFF" state (when medication is less effective), with a motor examination score of 33 or higher in the "OFF" state, and who are still able to walk without a walker or wheelchair.
- People who score above a certain threshold on a standard memory and thinking test (MoCA), with the minimum score depending on their level of education (confirm with trial site).
- People who are already on an optimised levodopa medication regimen — meaning they take levodopa at least three times a day at a total daily dose of at least 300 mg, combined with at least one other Parkinson's medication — but still experience poor symptom control or significant "wearing-off" periods, shown by a diary recording at least 2.5 hours of "OFF" time per day for three consecutive days during screening.
- People whose Parkinson's symptoms and medications have been stable for at least 4 weeks before screening; people also being treated for Gaucher disease must have been on a stable treatment for that condition for at least 3 months before screening.
- People who agree to use a highly effective method of contraception consistently from the screening period until at least one year after receiving the study treatment (applies to both men and women who are able to have children).
- Men who agree not to donate sperm, and women who agree not to donate eggs, from the screening period until at least one year after receiving the study treatment.
- People (or their legal guardian) who understand the trial information, its purpose, and its risks, and are willing to provide signed and dated consent for their health information to be used.
- People who have a reliable support person — such as a family member, friend, or carer — who is willing and able to attend study visits when needed and help provide information about the person's health, thinking, and physical abilities.
Who may not be able to join
Each point below is a reason the trial team may not be able to accept someone. It is not a list of requirements to meet.
- People who have an atypical or secondary form of parkinsonism caused by things such as brain injury, tumours, infections, stroke-related conditions, other neurological disorders, or exposure to certain drugs, chemicals, or toxins; or people with other serious neurological conditions the trial investigator considers would interfere with assessing the treatment's safety or effectiveness.
- People who currently have an active infection (including hepatitis B, hepatitis C, or syphilis), or who have had a serious infection such as pneumonia, blood poisoning, or a brain/spinal cord infection in the 12 weeks before screening.
- People who have had severe liver disease, severe immune system problems, or autoimmune disease in the 6 months before screening, or who need long-term medication to suppress their immune system.
- People with poorly controlled diabetes or high blood pressure that the investigator considers unsuitable for the study or likely to significantly affect the results.
- People who have had a stroke, a mini-stroke (TIA), unstable chest pain (angina), a heart attack, severe heart failure, or significant heart rhythm problems in the year before screening.
- People with a history of epileptic seizures or unexplained loss of consciousness that the investigator considers unsuitable for participation.
- People who have had severe allergic reactions, or who are known to be sensitive to any inactive ingredient in the study drug or to immunosuppressant medications required by the trial.
- People who have medical conditions that make the use of corticosteroids or sirolimus (medications used as part of the trial) inadvisable, including bone thinning with spinal fractures in the past year, poorly controlled high cholesterol or blood fats, kidney problems, or lung disease (confirm with trial site for full list).
- People who have been newly diagnosed with or have had unstable mental health conditions in the past year that may interfere with the trial, including severe confusion, severe depression (as measured by a standard rating scale), or thoughts of suicide or self-harm.
- People with a history of cancer in the 3 years before screening, except for certain fully treated and stable cancers such as some skin cancers, non-spreading prostate cancer, or carcinoma in situ that has been fully cured and stable for at least 6 months.
- People who have any other condition the investigator considers would make trial procedures unsafe or difficult to carry out, including conditions affecting the spine, bleeding disorders, blood clotting problems, low platelet counts, or raised pressure inside the skull.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
If you need support right now
This page includes language about suicide and self-harm, because that is how the trial's eligibility criteria are written. If reading it has been hard, you do not have to keep going. The services below are free, confidential, and answered by people trained for exactly this.
If you or someone else is in immediate danger, call 000.
- Lifeline — 24/7 crisis support: 13 11 14
- Suicide Call Back Service — 24/7 counselling: 1300 659 467
- 13YARN — 24/7, for Aboriginal and Torres Strait Islander people: 13 92 76
- Beyond Blue — 24/7 mental health support: 1300 22 4636
- Kids Helpline — 24/7, free and private for anyone aged 5 to 25: 1800 55 1800
- headspace — mental health support for 12 to 25 year olds: headspace.org.au
- Emergency: 000
Outside Australia? The numbers above are Australian and will not work from overseas. Find a Helpline is a free directory of crisis lines in over 130 countries, run by ThroughLine with the International Association for Suicide Prevention. If you are in immediate danger, call your local emergency number.
Voxsanity is not a crisis service, and nothing on this page is medical advice. More Australian services are listed on our support lines page.
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Trial details
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Data last synced from ClinicalTrials.gov: 24 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.