Clinical Trials2019-11-27T06:56:38+00:00

Clinical trials

This section of the AAN website will list clinical trials for amyloidosis patients once the first location or site has opened for patient screening and registration. Once a trial has been posted as being open at one site, all other sites that have been selected to participate for that particular trial will also be listed.

It will be indicated which clinical trial site has been “activated” or is “pending” to open. Once a clinical trial has closed for accrual at all sites it will be removed from the website.

The contacts for each of the clinical trials will be listed and please note that the clinical trial contacts are often different to the clinic contacts.

The clinical trials are subdivided into amyloidosis types: ATTR, AL and “Other”. This page is designed to be understandable for both patients and clinicians alike.

 This section of the AAN website will list clinical trials for amyloidosis patients.

The patient group, study title and short description including key inclusion and exclusion criteria will be included.

The trial status at each site will be indicated by a symbol.
The key is as follows:

Clinical trial key

Transthyretin Amyloidosis (ATTR) trials

This section includes clinical trials open (or about to open) for patients with either “wild type” (acquired) or hereditary transthyretin amyloidosis.

Occasionally, trials may only apply for patients with hereditary disease, or only “wild type” disease, so please take care to read the “Patient Group” information about the trial of interest.

Patient Group: Cardiac ATTR for any type of ATTR ie the trial is open to both wild type (ATTRwt) and hereditary ATTR (ATTRv) patients with cardiac involvement

Brief Description: An international Phase 3 “open label extension” trial for cardiac ATTR patients (both hereditary and wild-type cardiac ATTR) providing access to Tafamidis for up to 60 months or until access can be obtained via prescription (whichever happens first). All patients receive the active drug Tafamidis (ie. no-one receives placebo).

Key inclusion criteria: Evidence of cardiac transthyretin amyloidosis either from cardiac technetium PYP/DPD/HMDP scintigraphy scan or cardiac biopsy.

Key exclusion criteria: Liver and/or heart transplant, or implanted cardiac mechanical assist device.

Access Clinical Trials Database

Sites

New South Wales

Status:
Site: Westmead Hospital, Sydney
Email: mtay2576@uni.sydney.edu.au

Victoria

Status:
Site: Box Hill Hospital, Melbourne
Email: suzi.jakicic@monash.edu
Phone: 03 9094 9505

Queensland

Status:
Site: Princess Alexandra Hospital, Brisbane
Email: CindyHall@health.qld.gov.au
Phone: 07 3176 5145

Patient Group: Patients with any type of symptomatic ATTR cardiac amyloidosis. This trial therefore includes those with ATTRwt (wild-type) or ATTRv (hereditary) cardiac amyloidosis.

Brief Description: An international Phase 3 prospective randomised blinded study to evaluate the efficacy and safety of AG10 800 mg compared to placebo in subjects with symptomatic transthyretin cardiomyopathy. Patients will receive either AG10 or placebo.

The randomisation of AG10 versus placebo is 2:1 – this means that for every 3 patients, two will receive AG10 tablets, one will receive placebo. Your doctor will not know which tablet you are one. Those who complete 30 months of the trial will then be invited to receive guaranteed AG10 in an open label extension study.

AG10 is a TTR stabilizer – it belongs to the same group of pharmaceutical therapeutics as Tafamidis. In vitro studies (i.e. “in the test tube”) suggest that AG 10 may be more effective than Tafamidis, with stabilization of the TTR seen in 90-95% with AG10 versus 40-60% with Tafamidis. This trial is to determine its clinical efficacy and safety. Plans are underway such that this trial will be available in every capital city within Australia, as well as several key sites in New Zealand.

Key inclusion criteria: Symptomatic heart failure with an NT-proBNP > 300 ng/L

Key exclusion criteria: Age >90 years, NT-proBNP > 8000ng/L and eGFR <15mL/min/1.73 m2

Access Clinical Trials Database

Sites

New South Wales

Status:
Site: St Vincent’s Hospital, Sydney
Email: Clare.coates@svha.org.au
Phone: 02 8382 3073
Status:
Site: Westmead Hospital, Sydney
Email: mtay2576@uni.sydney.edu.au

Queensland

Status:
Site: Princess Alexandra Hospital, Brisbane
Email: CindyHall@health.qld.gov.au
Phone: 07 3176 5145

Victoria

Status:
Site: Box Hill Hospital, Melbourne
Email: suzi.jakicic@monash.edu
Phone: 03 9094 9505

Western Australia

Status:
Site: Fiona Stanley Hospital
Email: fsh.ahfcts@health.wa.gov.au

Tasmania

Status:
Site: The Royal Hobart Hospital
Contacts: to be notified

Patient Group: Inherited ATTR (ATTRv) with neuropathy

Brief Description: An international Phase 3 trial to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (ATTRv). Participants will receive the RNAi therapy vutrisiran (given subcutaneously every 3 months) or the reference (comparator RNAi) therapy patisiran (given intravenously every 3 weeks) during the initial treatment period. The initial treatment period is followed by a treatment extension period during which all participants in the patisiran group will switch to vutrisiran.

Key inclusion criteria: A diagnosis of inherited ATTR and reaching a certain level of neurologic impairment using the Neurologic Impairment Score (NIS)

Key exclusion criteria: Prior liver transplant or NYHA heart failure classification >2

Practical Point: All Australian patients need to be assessed by neurologists at one site (Westmead Hospital, Sydney) to determine eligibility for this trial as per trial requirements using rigorous clinical and neurologic testing methods.

As of 26th November 2019, this trial recruited all patients required for the study and enrollment has closed.

Access Clinical Trials Database

Sites

New South Wales

Status:
Site: Westmead Hospital, Sydney
Email: mtay2576@uni.sydney.edu.au

Queensland

Status:
Site: Princess Alexandra Hospital, Brisbane
Email – Dr Emad Abro: amyloiodosis@health.qld.gov.au

Victoria

Status:
Site: Box Hill Hospital, Melbourne
Email: suzi.jakicic@monash.edu
Phone: 03 9094 9505