KHI Current Project
Endpoints for Hyperoxaluria Clinical Trials
Primary Hyperoxaluria Patient Survey
Eligible patients with Primary Hyperoxaluria who are at least 13 years of age (or their proxy) are invited to participate in a brief online survey. The goal of this survey is to understand how PH affects people's lives and how we can work to find better treatments for it. Survey closes March 29.Survey
Primary hyperoxaluria and enteric hyperoxaluria are rare disorders that cause frequent kidney stones, nephrocalcinosis, and kidney failure. Promising new treatments are emerging to manipulate oxalate biosynthesis and absorption from the gastrointestinal tract. Careful evaluation of their effectiveness is essential for successful implementation. The major goal of this KHI project is to bring together patients, families, advocacy organizations (Oxalosis and Hyperoxaluria Foundation, OHF), clinicians, scientists, pharmaceutical companies, and the US Food and Drug Administration (FDA) to evaluate potential biochemical endpoints for use to establish efficacy of agents to treat hyperoxaluria to expedite their approval. Work will culminate in the authorship of a document that summarizes the consensus assessment.
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.
|Co-Chair||John C. Lieske, MD, FASN||Mayo Clinic|
|Co-Chair||Dawn S. Milliner, MD||Mayo Clinic|
|Member||Felicity Enders, PhD, MPH||Mayo Clinic|
|Member||W. Todd Lowther, PhD||Wake Forest School of Medicine|
|Board of Directors Liaison||Uptal D. Patel, MD||Gilead Sciences, Inc.|
|Staff Liaison||Kim Hollander||Oxalosis & Hyperoxaluria Foundation|
|Staff Liaison||Julie Bertarelli||Oxalosis & Hyperoxaluria Foundation|
|Staff Liaison||Meaghan Allain||Kidney Health Initiative|