Kidney Health Initiative (KHI)

KHI Current Project

Identification of Appropriate Endpoints for Clinical Trials in Hyperoxaluria

Patient Care Issue:

Primary hyperoxaluria and enteric hyperoxaluria are rare disorders. Patients typically experience frequent kidney stones, and can develop nephrocalcinosis, oxalate nephropathy, and end stage renal 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.

KHI Solution:

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.


Open Call for Workgroup Members

To apply to serve on this workgroup, please send the following materials in an email to with a subject "Hyperoxaluria Workgroup":

  1. Name and contact information
  2. CV or Resume
  3. Brief description of why you would like to participate (250 words or less)
  4. Brief description of what you will contribute (250 words or less)
  5. Note your interest in serving as chair, member, or either position



Timeframe for Completion

Fall 2018


John C. Lieske, MD, FASN
Mayo Clinic

Dawn S. Milliner, MD
Mayo Clinic


Felicity Enders, PhD, MPH
Mayo Clinic

W. Todd Lowther, PhD
Wake Forest School of Medicine

KHI Board of Directors Liaison:

Uptal D. Patel, MD
Gilead Sciences, Inc.

KHI Staff

Melissa West
KHI Project Director

OHF Staff

Kim Hollander
Executive Director

Julie Bertarelli
Operations Coordinator

Selected References:
  1. Tang X, Bergstralh EJ, Mehta RA, Vrtiska TJ, Milliner DS, Lieske JC. Nephrocalcinosis is a risk factor for kidney failure in primary hyperoxaluria. Kidney international. 2015;87(3):623-31.
  2. Zhao F, Bergstralh EJ, Mehta RA, Vaughan LE, Olson JB, Seide BM, et al. Predictors of Incident ESRD among Patients with Primary Hyperoxaluria Presenting Prior to Kidney Failure. Clinical journal of the American Society of Nephrology : CJASN. 2016;11(1):119-26.

If you have any questions, please do not hesitate to contact KHI at