KHI Workgroup Opportunity
Endpoints for Hyperoxaluria Clinical Trials
Overview
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. There has not been accepted endpoints to evaluate new therapeutic agents. The Kidney Health Initiative convened two projects to evaluate potential biochemical endpoints for use to establish efficacy of agents to treat hyperoxaluria to expedite their approval. These projects included patients, families, advocacy organizations (Oxalosis and Hyperoxaluria Foundation), clinicians, scientists, pharmaceutical companies, and the US Food and Drug Administration (FDA). Their work culminated in the authorship of the publications below that summarize the consensus assessments.
Deliverable
End Points for Clinical Trials in Hyperoxaluria: Case Study of Patient-Focused Drug Development in a Rare Disease- Identification of Endpoints for Clinical Trials in Primary Hyperoxaluria
A Kidney Health Initiative working group of physicians, scientists, primary hyperoxaluria patients, industry, and U.S. regulators critically examined the published literature for clinical outcomes and potential surrogate endpoints that could be used to evaluate new treatments.
Deliverable
Primary Hyperoxaluria The Patient and Caregiver Perspective
End Points for Clinical Trials in Primary Hyperoxaluria - Surrogate Endpoints for Clinical Trials in Enteric Hyperoxaluria
A multidisciplinary group convened by the Kidney Health Initiative reviewed the evidence supporting potential end points for clinical trials in enteric hyperoxaluria.
Deliverable
Pathophysiology and Treatment of Enteric Hyperoxaluria
End Point Considerations for Clinical Trials in Enteric Hyperoxaluria