Clinical trials

Welcome to the Amyloidosis Clinical Trials page!

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 Light Chain Amyloidosis (AL) trials

The “CAEL-101” monoclonal antibody trials for newly diagnosed AL Amyloidosis with cardiac involvement are now open in Australia!

All four AAN services, as well as the Royal Adelaide Hospital, are now recruiting patients to these studies!

CAEL101-301 and CAEL101-302 Trials

CAEL101-301: A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis

CAEL101-302: A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIa AL Amyloidosis – A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

What is the drug in question?
CAEL-101 is a monoclonal antibody that attaches itself to part of the AL amyloid fibril in your tissues, and makes it easier for your own immune system to clear the amyloid deposits.

Who is eligible for the trial?
Patients with symptomatic newly diagnosed AL cardiac amyloidosis are eligible for these trials.
Patients who have already had treatment for their AL amyloidosis, or those without significant heart involvement are not eligible/
Does every patient receive the actual trial drug?
These are international Phase 3 prospective randomised placebo-controlled double-blinded studies.
All patients will receive the normal treatment for new diagnosed AL amyloidosis, that being bortezomib (Velcade), cyclophosphamide and dexamethasone.
In addition, patients will be randomized 2:1 to also receive either the CAEL-101 infusion delivered through an IV line into your vein (a “drip”) or the placebo (“dummy” drug) infusion. That means you have a 2 out of 3 chance of receiving the active drug. Neither you nor your doctor will know whether you are on the active drug or the placebo.

How is the drug administered?
CAEL-101 is administered as a intravenous (“a drip into a vein”) infusion. Patients will receive their injection weekly for four weeks then fortnightly.

How long does the trial go for?
At least 50 weeks (almost a year).

Anything else I need to know?
As part of the assessment for the trial, you will need to undergo several 6 minute walking tests, echocardiography (heart ultrasounds), and complete quality of life questionairres.

Key inclusion criteria:
Systemic AL (not ATTR) amyloidosis, involving the heart, as proven by

Biopsy proof for amyloid deposits (such as bone marrow, kidney or heart biopsy)
Cardiac MRI and/or echocardiography demonstrating signs of cardiac amyloidosis
Elevated serum free light chains >40mg/L.
NT-proBNP >650ng/L

Key exclusion criteria:

Patients who have received previous therapy for AL Amyloidosis
Patients whose systolic blood pressure if <90mmHg
Patients on dialysis, or taking high doses of prednisolone or doxycycline.

For more information, please check the five recruiting centres listed below and the Clinical Trials Gov website:

Sites

Queensland

Status:
Site:	Queensland Amyloidosis Centre, Princess Alexandra Hospital, Brisbane
Contact:	A/Prof Peter Mollee Email: amyloidosis@health.qld.gov.au

Victoria

Status:
Site:	Victorian and Tasmanian Amyloidosis Centre, Eastern Health, Melbourne
Contact:	Simon Gibbs simon.gibbs@monash.edu

South Australia

Status:
Site:	Royal Adelaide Hospital, Adelaide
Contact:	Dr Noemi Horvath Email:TBC

New South Wales

Status:
Site:	Westmead Hospital, Sydney
Contact:	Dr Fiona Kwok Email: WSLHD-TTRtrials@health.nsw.gov.au

Western Australia

Status:
Site:	Fiona Stanley Hospital, Perth
Contact:	Dr Hasib Sidiqi Email: coral.almeida@health.wa.gov.au Hasib.Sidiqi@health.wa.gov.au

Please check this website weekly for updates!

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Transthyretin Amyloidosis (ATTR) trials

The “Cardio TTRansform” trial for ATTR Amyloidosis is now open in Australia!

Please click on the links below for more details.

If the Cardio TTRansform study isn’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) – available at all AAN services
Doxycycline and/or
EGCG (green tea extract).

CARDIO-TTRansform

A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

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 patient 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.

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