Clinical trials
Welcome to the Clinical Trials page of the Australian Amyloidosis Network website!
This page is designed to be easy to understand for both patients and clinicians alike.
Clinical trials are subdivided into major amyloidosis types: ATTR, AL and Other.
Please SCROLL DOWN for full details.
Clinical trials are listed here once the first health service in Australia has fully opened the trial for patient enrollment.
Once a trial has been listed as being open (or “activated“) at one site, other sites in Australia selected to participate will also be listed, even thought they may not yet be fully open (“pending“).
Please note : Listing a clinical trial on this website does not necessarily equate as endorsement of the trial by the Australian Amyloidosis Network. Any patient interested in a trial should discuss the pros and cons of the studies with their treating practitioner.
Welcome to the Clinical Trials page of the Australian Amyloidosis Network website!
This page is designed to be easy to understand for both patients and clinicians alike.
Clinical trials are subdivided into major amyloidosis types: ATTR, AL and Other.
Please SCROLL DOWN for full details.
Clinical trials are listed here once the first health service in Australia has fully opened the trial for patient enrollment.
Once a trial has been listed as being open (or “activated“) at one site, other sites in Australia selected to participate will also be listed, even thought they may not yet be fully open (“pending“).
Please note : Listing a clinical trial on this website does not necessarily equate as endorsement of the trial by the Australian Amyloidosis Network. Any patient interested in a trial should discuss the pros and cons of the studies with their treating practitioner.
For each trial, a description of the trial, including key inclusion and exclusion criteria, and local contacts are provided.
The trial status at each site will be indicated by a symbol.
The key is as follows:

Systemic AL Amyloidosis trials
Currently, there are no actively recruiting trials in AL amyloidosis, although several are in set-up and hope to open by Easter 2021.
Please keep checking this website regularly for updates!
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Transthyretin Amyloidosis (ATTR) trials
Three new ATTR trials have opened in Australia, with more sites opening!
Please click on the links below for more details.
If these trials aren’t right for you or not available in your area, there are still treatments for ATTR readily available off-trial that your doctor can discuss with you. These include:
- Diflunisal (similar to tafamidis)
- Doxycycline and/or
- EGCG (green tea extract).
What is the drug in question?
AKCEA-TTR-LRx is a gene-silencing therapy, designed to reduce the production of TTR by the liver, and thus slow or stop the formation and progression of TTR amyloidosis.
Who is eligible for the trial?
Patients with symptomatic ATTR cardiac amyloidosis are eligible for this trial.
Both ATTRwt (wild-type) and ATTRv (hereditary) disease groups are included.
Does every patint receive the actual trial drug?
This is an international Phase 3 prospective randomised placebo-controlled double-blinded study.
Patients will be randomized 1:1 (50;50 chance) to receive either the AKCEA-TTR-LRx injection or the placebo (“dummy” drug) injection. Neither you nor your doctor will know whether you are on the active drug or the placebo.
How is the drug administered?
AKCEA-TTR-LRx is administered as a subcutaneous (“under the skin”) injection. Patients will receive their injection every four weeks
How long does the trial go for?
The TTRansform trial lasts 120 weeks (27 months).
Anything else I need to know?
Patients can also take EGCG and/or tafamidis during the study, but not diflunisal or doxycycline.
N.B. Neither EGCG nor Tafamidis will be supplied by the drug company, and tafamidis is currently not available on the PBS.
Key inclusion criteria:
1. “Symptomatic” heart failure defined as requiring hospitalisation or current/prior diuretic management.
2. NT-proBNP 600-6000 ng/L.
Key exclusion criteria:
1. Monoclonal gammopathy of undetermined significance (MGUS) unless fat, bone marrow, or heart biopsy confirms the absence of AL amyloid by mass spectrometry or immunoelectron microscopy.
2. eGFR <30mL/min/1.73 m2
3. Prior liver or heart transplant.
Access Clinical Trials Database
Sites
New South Wales
Status: | |
Site: | Liverpool Hospital, Sydney |
Contact: | Study co-ordinator: Natalia Inness: natalia.inness@health.nsw.gov.au Principal Investigator: Gayathri.Kumarasinghe@health.nsw.gov.au Phone: (02) 8738 3078 Fax: (02) 8738 3054. |
Status: | |
Site: | Westmead Hospital, Sydney |
Contact: | Dr Mark Taylor |
Email: | WSLHD-TTRtrials@health.nsw.gov.au |
Victoria
Status: | |
Site: | The Victorian and Tasmanian Amyloidosis Service, Box Hill Hospital, Melbourne |
Email: | simon.gibbs@monash.edu |
Phone: | 03 9094 9505 |
South Australia
Status: | |
Site: | Flinders Medical Centre, Adelaide |
Contact: | Fiona Wollaston, Study Coordinator, |
Email: | fiona.wollaston2@sa.gov.au |
Queensland
Status: | |
Site: | Princess Alexandra Hospital, Brisbane |
Contact: | Cindy Hall, Study Co-ordinator |
Phone: | 07 3176 5145 |
Western Australia
Status: | |
Site: | Genesis Care, Perth |
Contact: | Dr Peter Purnell |
Email: | cardiology.research@genesiscare.com |
What is the drug in question?
Patisiran is a gene-silencing therapy, designed to reduce the production of TTR by the liver, and thus slow or stop the formation and progression of TTR amyloidosis.
Who is eligible for the trial?
Patients with symptomatic ATTR cardiac amyloidosis are eligible for this trial.
Both ATTRwt (wild-type) and ATTRv (hereditary) disease groups are included.
Does every patint receive the actual trial drug?
This is an international Phase 3 prospective randomised placebo-controlled double-blinded study.
Patients will be randomized 1:1 (50;50 chance) to receive the patisiran infusion or the placebo (“dummy” drug) infusion for 12 months. Neither you nor your doctor will know whether you are on the active drug or the placebo.
After the first 12 months, all patients are guaranteed to receive the active patisiran drug for the following 12 months.
How is the drug administered?
Patisiran (or the placebo) is administered as an IV infusion (“into a vein”). Patients will receive their infusion every four weeks
How long does the trial go for?
The Apollo-B trial lasts 2 years – for the first 12 months, it is a 1:1 placebo controlled trial. then the following 12 months, all patients will receive patisiran.
Anything else I need to know?
Patients cannot take diflunisal, EGCG, doxycycline or tafamidis during the study, however, patients CAN take diflunisal in the second 12 months of the study with the open label patisiran.
Key inclusion criteria:
1. Diagnosis of cardiac ATTR by DPD or PYP cardiac amyloid bone scan. In the presence of monoclonal gammopathy of uncertain significance, a diagnosis of ATTR must be confirmed on biopsy using immunohistochemistry or mass spectrometry.
2. History of heart failure defined as requiring hospitalisation or with evidence of heart failure that currently requires diuretic management.
3. Tafamidis naïve or with progression on Tafamidis 4. NT-proBNP 600-8500ng/L.
Key Exclusion Criteria:
1. NYHA Class III and ATTR amyloidosis Stage 3 (defined as both NT=proBNP>3000ng/L and eGFR <45mL/min/1.73)
2. NYHA Stage IV
3. Polyneuropathy disability Score IIIa, IIIb or IV (requires cane or stick to walk or wheelchair bound).
4. eGFR < 30mL/min/1.73.
5. Concurrent diflunisal (30 day wash-out prior to dosing).
Access Clinical Trials Database
Sites
New South Wales
Status: | |
Site: | Westmead Hospital, Sydney |
Email: | WSLHD-TTRtrials@health.nsw.gov.au |
Queensland
Status: | |
Site: | Princess Alexandra Hospital, Brisbane |
Contact: | Cindy Hall, Study Coordinator |
Phone: | 07 3176 5145 |
Victoria
Status: | |
Site: | The Victorian and Tasmanian Amyloidosis Service, Box Hill Hospital, Melbourne |
Email: | simon.gibbs@monash.edu |
Phone: | 03 9094 9505 |
What is the drug in question?
Vutrisiran is a gene-silencing therapy, designed to reduce the production of TTR by the liver, and thus slow or stop the formation and progression of TTR amyloidosis.
Who is eligible for the trial?
Patients with symptomatic ATTR cardiac amyloidosis are eligible for this trial.
Both ATTRwt (wild-type) and ATTRv (hereditary) disease groups are included.
Does every patint receive the actual trial drug?
This is an international Phase 3 prospective randomised placebo-controlled double-blinded study.
Patients will be randomized 1:1 (50;50 chance) to receive either the Vutrisiran injection or the placebo (“dummy” drug). Neither you nor your doctor will know whether you are on the active drug or the placebo.
How is the drug administered?
Vutrisiran (or the placebo) is administered as a subcutaneous (“under the skin”) injection. Patients will receive their injection every three months.
How long does the trial go for?
The Helios-B trial lasts 3 years.
Anything else I need to know?
Patients can also take tafamidis after the first 12 months of the study, but they cannot take diflunisal, EGCG or doxycycline.
N.B. Tafamidis is currently not available on the PBS.
Access Clinical Trials Database
Key Inclusion Criteria:
1. Diagnosis of cardiac ATTR by DPD or PYP cardiac amyloid bone scan. In the presence of monoclonal gammopathy of uncertain significance, a diagnosis of ATTR must be confirmed on biopsy using immunohistochemistry or mass spectrometry.
2. History of heart failure defined as requiring hospitalisation or with evidence of heart failure that currently requires diuretic management.
2. Tafamidis naïve 3. NT-proBNP 600-8500ng/L.
Key Exclusion Criteria:
1. NYHA Class IV heart failure or NYHA Class III and ATTR amyloidosis Stage 3 (defined as both NT=proBNP>3000ng/L and eGFR <45mL/min/1.73)
2. NYHA Stage IV
3.Polyneuropathy disability Score IIIa, IIIb or IV (requires cane or stick to walk or wheelchair bound)
4. eGFR < 30mL/min/1.73m2
5. Prior organ transplant.
Sites
New South Wales
Status: | |
Site: | Westmead Hospital, Sydney |
Email: | WSLHD-TTRtrials@health.nsw.gov.au |
Status: | |
Site: | St Vincent’s Hospital, Sydney |
Email: | Carmen.Herrera@svha.org.au |
South Australia
Status: | |
Site: | Royal Adelaide Hospital |
Contact: | Denise Healy, Research Assistant for Dr Julie Bradley |
Email: | Denise.Healy@sa.gov.au. |
Tasmania
Status: | |
Site: | Royal Hobart Hospital |
Email: | david.russell@ths.tas.gov.au |
Queensland
Status: | |
Site: | Princess Alexandra Hospital, Brisbane |
Phone: |
07 3176 5145 |
Victoria
Status: | |
Site: | Cardiology Unit, Alfred Health |
Email: | TBA |
Phone: |