RESEARCH

Clinical trials

Clinical Trials

Do you have Alport Syndrome?

A new Australian clinical trial might be right for you.

‘Dietary Patterns in Kidney Disease: A longitudinal cohort study’.

If you are 18 years or older and have a Genetic Kidney Disease, we invite you to participate in a research study that aims to explore what people with Genetic Kidney Diseases are eating, and whether diet is related to disease progression.

The study is called ‘Dietary Patterns in Kidney Disease: A longitudinal cohort study’. This study is being conducted by researchers at the University of Wollongong and clinicians across hospitals in Australia and New Zealand.

If you are eligible, and choose to participate in this research, you will receive a $50 voucher at the start and end of the study.

Visit the KIDGEN patients page for more clinical research information.

Monash Health Hospital– ELOXX Clinical Trial

Monash Health Hospital is looking for small group of Alport patients aged 6-30 with certain gene changes (nonsense mutation) to participant in a clinical trial of testing a new treatment.

Eloxx company is testing a new treatment that they think might help certain people with Alport Syndrome. Alport syndrome is caused by changes (called mutations) in certain genes. Only a very small proportion of gene changes (called ‘nonsense mutations’ which account for approximately 3-5% that cause Alport syndrome) are potentially treatable by this new testing treatment (called ELX-02). The main goal of this study (called EL-014) is to learn whether ELX-02 is safe in Alport patients and whether it improves kidney function and hearing. The study will soon open trial sites in the UK and Australia.

Please note that the study team will need to know your genetic test result and latest kidney function reading (called eGFR) to be able to tell whether you might be eligible for this study.

Details of sites running the Eloxx clinical trial:

Australia – Monash Health, Melbourne (adults) – Prof. Kerr – Clinresneph@monashhealth.org.

Note: Criteria for people appropriate for the Eloxx study.

The study will be performed in Australia and UK and is looking for male and female participants who:

Are 6-30 years of age (inclusive)
Have a confirmed diagnosis of X-linked Alport syndrome with a nonsense mutation in COL4A5, or a diagnosis of autosomal recessive Alport syndrome with two nonsense mutations in either COL4A3 or COL4A4
Are on a stable dose of an ACE inhibitor or ARB for 4 weeks or more
Have over a certain value of kidney filtration rate (eGFR >60ml/min/1.73m2)
Have not received a kidney transplant
Live in UK and Australia or willing to travel to those countries
Available for 60 days treatment with bi-weekly visits, followed by 3 months with monthly visits

For more information email Professor Kerr: Clinresneph@monashhealth.org.

HERA Clinical Trial

Are you or someone you know living with Alport Syndrome? If you have been diagnosed with Alport Syndrome, you may be eligible to participate in a clinical trial investigating a new drug for adult patients with Alport Syndrome.

The clinical trial will look to see how well a new experimental drug works in preserving kidney function. This treatment is only available to patients participating in the clinical trial.

You may be eligible for this trial if:

You are aged 18-55 years old
You have confirmed Alport Syndrome via genetic testing
You have kidney disease
You do not have diabetes
You are less than 110kg

TO LEARN MORE PLEASE CONTACT:

Donna North: Royal Melbourne Hospital, Victoria, Australia.

email: donna.north@mh.org.au

Belinda Elford: Royal Brisbane & Womens Hospital, Queensland, Australia.

email: belinda.elford@health.qld.gov.au

Aron Chakera. Sir Charles Gairdner Hospital, WA, Australia.

Phone: aron.chakera@health.wa.gov.au

REATA Global Phase 2/3 Trial in CKD caused by Alport syndrome

Reata believes bardoxolone methyl has the potential to address the underlying causes of GFR loss in Alport syndrome patients because it activates molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting ROS-mediated pro-inflammatory signaling. These anti-inflammatory and tissue-protective effects suppress multiple cellular processes that conspire to promote GFR loss. Bardoxolone methyl and closely related structural analogs have been shown to improve renal function, reduce inflammation, and prevent injury, remodeling, and fibrosis in many animal models of renal injury and disease.

Bardoxolone methyl has been tested in seven studies of patients with CKD caused by type 2 diabetes that enrolled approximately 2,600 patients. These studies included a randomized, placebo-controlled 52-week Phase 2b study (“BEAM”) and a large, multinational Phase 3 study (“BEACON”) that enrolled only patients with severe (Stage 4) CKD. In these studies, bardoxolone methyl treatment significantly increased eGFR and creatinine clearance, significantly reduced uremic solutes (BUN, uric acid, and phosphate) in inverse correlation to eGFR increases, and numerically reduced renal SAEs and ESRD events.