Clinical trials

All trial sites were notified on the 10th June that the tafamidis access trial is now closed and that no further enrolments can occur. The pharmaceutical company who developed tafamidis explained in a letter to trial sites that this was to “concentrate efforts on achieving access though the Pharmaceutical Benefits Scheme (PBS)”.

It can sometimes take a long time to prepare a submission, negotiate price and list medications on the PBS. In the meantime, the AAN  is engaging with the pharmaceutical company to continue to explore all timely and feasible tafamidis access options.

Patient Group: Cardiac ATTR for any type of ATTR, meaning that 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.

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Sites

New South Wales

Victoria

Queensland

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

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Sites

New South Wales

Queensland

Victoria

Western Australia

Tasmania

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

Queensland

Victoria