This study will look at how well a drug that reduced the amount of oxalate in the body works in patients that have kidney disease and need dialysis treatment. People with kidney disease often have higher levels of oxalate in the blood. People with kidney disease are also at higher risk of having heart attacks, heart disease and strokes (these are called cardiovascular diseases). It is thought that high oxalate levels may increase the risk of these diseases. This study will investigate if this medicine can lower the amount of oxalate in the blood of dialysis patients and see if there is any change in the health of their heart. This medicine is already used for people who have high oxalate levels because of a genetic cause and has been used safely for patients on dialysis.
The study will put the participants randomly into either the group getting the study medicine or the group getting a placebo (this will be a solution of saline water). Neither participants not the doctors will know whether the drug or placebo is given until after the end of the study.
At the start of the study all the participants will have an echocardiogram (an ultrasound of the heart) and again 6 months later at the end of the study. We will also take blood tests once a month when the participants come for dialysis.
Male or female patients
Aged between 18 and 80 years old at the start of the study.
Women of child-bearing potential to consent to either abstinence or the use of contraception during the study period
Patients must have capacity to give written, informed consent to participate in the study prior to commencing the study. They must be fully aware of the aims, nature, planned interventions and potential risks of participating in the study. This consent must be obtained by the time of participant inclusion.
Known diagnosis of PH1, 2 or 3; or a pathological mutation documented to cause primary hyperoxaluria.
Established on haemodialysis for less than 2 months.
On peritoneal dialysis.
Combined haemodialysis and peritoneal dialysis.
Parallel
Lumasiran
DrugSubcutaneous injection, given as three monthly loading doses followed by one further maintenance dose.
0.9% Sodium Chloride (placebo)
DrugPlacebo, subcutaneous injection, given as three monthly loading doses followed by one further maintenance dose.
Pre-dialysis Plasma Oxalate concentration
The primary endpoint is the percentage change in pre-dialysis plasma oxalate levels in non-primary hyperoxaluria patients with raised plasma oxalate levels who are receiving maintenance haemodialysis.
Absolute change in pre-dialysis mean plasma
Absolute change in pre-dialysis mean plasma oxalate levels from baseline to month 3-6, comparing the treatment group with the placebo group.
Side effects (tolerability)
Tolerability will be assessed using an adapted FACIT Instrument validated questionnaire (severity of side effects rated on scale between 0 and 4). Again, the answers of the two study arms will be compared.
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Charite University, Berlin, Germany
Lead sponsor
Alnylam Pharmaceuticals
Collaborator
