Endpoints for Hyperoxaluria Clinical Trials
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: The Patient and Caregiver Perspective