Clinical Trial Endpoints

Patient-Reported Outcome Measures for Novel Renal Devices

Attention to the innovation deficit in treatments for kidney failure is increasing. As development of next generate kidney replacement therapy begins to accelerate, the perspectives of people with kidney failure must not be overlooked. To bridge this gap, the Kidney Health Initiative published a conceptual framework for patient reported outcomes regulators and device developers can use to incorporate the patient perspective into the total product lifecycle. A workgroup convened to review exiting patient-reported outcome measures (PROMs), synthesize existing data on patient preferences, and develop a framework for future PROMs that could be used in the evaluation of transformative renal replacement therapy devices. Such a framework can be used to catalyze patient-centered innovation by incorporating patient perspectives into both the development and regulatory evaluation stages of the total product life cycle.

Toward Patient-Centered Innovation 


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.

Endpoints for Clinical Trials in Primary Hyperoxaluria 


Surrogate Endpoints in IgA Nephropathy

IgA nephropathy, the most common glomerular disease worldwide, may progress to kidney failure over many years. There are no approved treatments and no accepted surrogate endpoints for registration of investigational products for this disease. The goals of this project are to evaluate and identify surrogate endpoint(s) for use in clinical trials in patients with different risks of progression to kidney failure. The workgroup, representing all KHI stakeholders, will critically analyze data from the literature, available patient registries and clinical trials and critically evaluate candidate surrogate endpoints according to the general criteria considered by the Food and Drug Administration.

Proteinuria Reduction as a Surrogate End Point in Trials of IgA Nephropathy 


Outcome Measures in Lupus Nephritis Trials

In partnership with the Lupus Nephritis Trial Network, a workgroup analyzed existing data to test for clear, valid endpoints for lupus nephritis trials. The workgroup contacted pharmaceutical companies that have completed trials in lupus nephritis to discuss the inclusion of their data in this project. Recognizing the value of including physicians treating these patients, the workgroup recruited global cohort sites to participate in the project.

Upon completion of the data analysis, the workgroup recommended a core set of outcome measures, biomarkers, surrogate markers, and clearly defined terms that should be incorporated into all future lupus nephritis trials. Developing these measures will make it much more feasible for companies to invest in the research necessary to improve treatments for patients with this serious disease.

Establishing Surrogate Kidney Endpoints for Lupus Nephritis Clinical Trials: Development and Validation of a Novel Approach to Predict Future Kidney Outcomes 


Clinical Trial Endpoints for Dialysis Vascular Access

This project convened diverse stakeholders (health professionals, industry, dialysis organizations, and federal agencies) to clarify appropriate trial endpoints for vascular access trials best suited to inform clinical, regulatory, and coverage decisions, in those circumstances where clinical data is required to support the decisions. The workgroup developed parallel white papers on catheters, fistulas, and grafts endpoints. Learn more about this project here.

Clinical Trial End Points for Hemodialysis Vascular Access FDA Regulatory Perspectives for Studies on Hemodialysis Vascular Access Recommended Clinical Trial End Points for Dialysis Catheters Definitions and End Points for Interventional Studies for Arteriovenous Dialysis Access