Primary hyperoxaluria and enteric hyperoxaluria are rare disorders. Patients typically experience frequent kidney stones, and can develop nephrocalcinosis, oxalate nephropathy, and kidney failure. There are no approved pharmacologic therapies, and available treatment options are non-specific and of limited effectiveness. Patients with severe primary hyperoxaluria are currently treated with liver transplantation, an extreme intervention given that liver function is otherwise normal. New treatments are sorely needed.

Given the slowly progressive and unpredictable rate of decline in kidney function in most patients, decrease in kidney function or end-stage renal disease is not a feasible endpoint for clinical trials. Kidney stones are common, but the stone events are also not a practical endpoint for clinical trials as their incidence is unpredictable, and reliable identification of stone events is cumbersome, potentially unsafe (as in the case of radiation exposure with computed tomography, the gold standard), and is not standardized. Thus, it is important to identify other endpoint(s) that can be reliably measured and can demonstrate a beneficial treatment effect within a relatively short timeframe in order to expedite the approval of drugs for the treatment of both primary and secondary hyperoxaluria.

The major goal of this KHI project is to bring together the community of patients, families, advocacy organizations (OHF), clinicians, scientists, pharmaceutical companies and the FDA to evaluate potential biochemical endpoints that could be used to establish the efficacy of therapeutic agents for the treatment of primary and secondary forms of hyperoxaluria, and expedite their approval. This project will culminate in the authorship of a document that summarizes the consensus assessment.