Previously active trials
All Types of Amyloid
None currently
Systemic Light Chain Amyloidosis (AL) trials
- A Single Arm, Open-Label, Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis. The first part of the study is phase 1 dose-escalation and the second part will be phase 2.
- An orally bioavailable inhibitor of the anti-apoptotic protein B-cell lymphoma 2(Bcl-2), with potential pro-apoptotic and antineoplastic activities. Upon oral administration, Bcl-2 inhibitor ZN-d5 targets, binds to and inhibits the activity of Bcl-2.
- A Phase 3 Randomized, MultI-Centre Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs Placebo Plus Standard of Care in Newly Diagnosed Mayo Stage IV Subjects with Light Chain AL Amyloidosis
- Birtamimab is a humanized monoclonal antibody being investigated as a treatment for amyloid light chain (AL) amyloidosis and potentially AA amyloidosis.
A Phase Ib/II study of Venetoclax, IBERdomide and dexamethasone for patients in first or
second relapse of Multiple myeloma and/or systemic AL amyloidosis with t(11;14)
What is the drug in question?
Iberdomide is a “CelMod”, which is similar to lenalidomide and pomalidomide. It is well tolerated with proven activity in myeloma.
It is hoped that iberdomide to be more effective and less toxic than lenalidomide in the treatment of AL amyloidosis.
Venetoclax, a BCL2 inhibitor, which used in many haematological conditions, but in myeloma and AL amyloidosis, venetoclax specifically targets the effects of t(11;14) mutation.
Who is eligible for the trial?
- Patients with AL amyloidosis who have relapsed after 1 or 2 lines of treatment previously.
- Patients must have have the t(11;14) translocation (genetic variation) detected on their bone marrow biopsy (seen in ~50% of all AL amyloidosis patients).
- Patients must have serum free light chains (SFLC) >100mg/L to qualify
How is the drug administered?
This clinical trial is designed to see if the combination of both medications with the steroid, dexamethasone, has very high response rate, has quicker response times and is well tolerated.
This is an Australian only study of 50 patients.
Note that this treatment is an all oral (tablet) treatment.
You will be given medication to minimise the risk of these side-effects.
How long does the trial go for?
The three medications will continue until it stops working or if the patient has significant sideeffects and/or wants to withdraw from the study.
While this combination is expected to be well tolerated, the more common potential sideeffects of this treatment are low blood counts, rash, infections and clots.
Sites
Victoria
| Status: | |
| Site: | Eastern health (Box Hill Hospital), Melbourne. |
| Contact: | Olga Motorna and Stephen Ting Email: olga.motorna@easternhealth.org.au |
| Status: | |
| Site: | St Vincent’s Hospital, Melbourne. |
| Contact: | Shirlene Sim and Hang Quach Email: shirlene.sim@svha.org.au |
Queensland
| Status: | |
| Site: | Townsville University Hospital |
| Contact: | Hock Choong Lai Email: hock.lai@health.qld.gov.au |
New South Wales
| Status: | |
| Site: | Gosford Hospital |
| Contact: | Cecily Forsyth Email: cecily.forsyth@healthmail.com.au |
| Status: | |
| Site: | Royal North Shore, Sydney |
| Contact: | Ian Kerridge Email: ian.kerridge@health.nsw.gov.au |
| Status: | |
| Site: | Liverpool Hospital |
| Contact: | Adam Bryant Email: adam.bryant1@health.nsw.gov.au |
South Australia
| Status: | |
| Site: | Royal Adelaide Hospital |
| Contact: | Elizabeth Tucker Email: elizabeth.tucker@sa.gov.au Angie Yong Email: Angie.Yong@sa.gov.au |
Transthyretin Amyloidosis (ATTR) trials
What is the drug in question?
ALXN2220 is a recombinant human anti-ATTR antibody that was developed for the removal of ATTR by phagocytic immune cells.
What does the study hope to prove?
Efficacy of ALXN2220 in the treatment od ATTR cardiomyopathy.
Who is eligible for the trial?
Male or Female between 18 and 90 years of age with confirmed ATTR-CM.
Does every patient receive the actual trial drug?
Randomisation is in 2:1 ratio to receive either study drug (2/3) or placebo (1/3).
How is the drug administered?
By intravenous infusion every 4 weeks.
How long does the trial go for?
48 months
Key inclusion criteria:
Subjects with confirmed ATTR-CM, history or current heart failure (class 2-4 NYHA dyspnoea) and elevated cardiac biomarkers (NTproBNP).
Key exclusion criteria:
- Other non TTR types of amyloidosis
- Cardiomyopathy caused by other condition
- Recent (within 3 months) acute CV event
- Low LV ejection fraction (<30%)
- Severe non cardiac medical illness (lung, liver, kidney)
- Malignancy within last 5 years
- Solid organ recipient
- Low body weight (<40kg)
Where to go for more information:
See below.
Sites
Victoria
| Status: | |
| Site: | Eastern health (Box Hill Hospital), Melbourne |
| Status: | |
| Site: | The Alfred, Melbourne |
Queensland
| Status: | |
| Site: | Princess Alexandra Hospital, Brisbane |
| Contact: | Amyloidosis@health.qld.gov.au |
New South Wales
| Status: | |
| Site: | Westmead Hospital, Sydney |
| Contact: | WSLHD-TTRtrials@health.nsw.gov.au |
| Status: | |
| Site: | St Vincent’s Hospital, Sydney |
Western Australia
| Status: | |
| Site: | Joondalup, Perth |
South Australia
| Status: | |
| Site: | Flinders Medical Centre |
| Contact: | Name: Fiona Wollaston Email: fiona.wollaston2@sa.gov.au |
- This is a Phase 2 open-label extension study to evaluate the long-term safety, tolerability, and clinical activity of AT-02.
- AT-02 is a therapeutic candidate developed by Attralus for treating systemic amyloidosis. It’s a fusion protein consisting of a pan-amyloid removal (PAR) peptide technology and a humanized IgG1 antibody. The PAR peptide binds to all types of amyloid and delivers the antibody to the amyloid deposits, stimulating the immune system to remove the amyloid.
- A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM).
- NI006 is a recombinant human anti-ATTR antibodythat was developed for the removal of ATTR by phagocytic immune cells.
What is the drug in question?
NTLA-2001- clustered regularly interspaced short palindromic repeats (CRISPR) of RNAs encoding protein 9 (Cas9) formulated in lipid nanoparticle for IV use.
What does the study hope to prove?
Safety and efficacy of editing (removing) gene responsible for TTR synthesis in patients with hereditary ATTR with polyneuropathy.
Who is eligible for the trial?
Subjects with hereditary ATTR with polyneuropathy.
Does every patient receive the actual trial drug?
No. Half of them (50%) will receive placebo. However, after 12-18 months (depending on rate of any progression in nerve disease) participants may choose to receive blinded crossover treatment (either placebo or NTLA-2001) ie what ever they did not receive on the trial initially .
How is the drug administered?
Single intravenous infusion
How long does the trial go for?
This depends on whether the participant chooses to receive the blinded crossover treatment after 12-18 months.
Key inclusion criteria:
Male and Female aged 18 to 85 years old inclusive with hereditary ATTR with polyneuropathy who signed the informed consent who are naive to TTR silencers (siRNAs and ASOs).
Key exclusion criteria:
- Other known causes of sensorimotor or autonomic neuropathy
- Amyloidosis due to a non-TTR protein
- Known leptomeningeal amyloidosis
- Known diabetes mellitus
- Recent (within 3 months) cardiac (heart attack) and non-cardiac (stroke, PE, DVT) event
- End stage liver disease
- Active infection
- Malignancy within preceding 3 years
- Prior receipt of TTR silencer (siRNA or ASO)
- Solid organ recipient
Where to go for more information:
See below.
Sites
New South Wales
| Status: | |
| Site: | Westmead Hospital, Sydney |
| Contact: | WSLHD-TTRtrials@health.nsw.gov.au |
