KHI Workgroup Opportunity

Clinical Trials in Pediatric Patients with IgA Nephropathy (IgAN)

Community Unmet Need

In recent years, there has been a dramatic increase in drug development for IgAN; however, clinical trials are largely being conducted in adults. At this time, it is not clear whether the efficacy and safety of these treatments should also be assessed in children with IgAN, and, if so, how best to design studies to obtain the data needed to support use of novel therapies in this population.

Project Approach

The project team will approach this project by engaging subject matter experts to assess the current literature and trial landscape to formulate a community consensus on a path forward for developing therapies for children with IgA Nephropathy. Additionally, the project team will engage stakeholders who have clinical trial or registry data that can be used to inform natural history of disease research, or identify biomarkers candidates and outcome measurements that advance therapies for children living with IgA Nephropathy. This data would then be shared to a central repository for analysis in potential partnership of organizations such as the Critical Path Institute. This project is divided into two phases:

  1. Develop a harmonized data set for pediatrics with IgA Nephropathy
  2. Publish a report of findings of data accumulation/analysis from shared registry and trial data and suggest a path forward for developing therapies for children with IgA Nephropathy.


  1. Harmonized dataset for pediatrics with IgA Nephropathy
  2. Manuscript

Workgroup Applications

KHI invites interested stakeholders who have direct experience and/or expertise in IgAN in the pediatric population to become a member of this project team. Of particular interest are experts representing the following communities:

  • Industry Representatives Developing Therapies for IgAN
  • Researchers/Clinicians
  • Patients or Patient Organizations
  • Registry Groups
  • Statisticians

If you are interested in serving on this project team, please email KHI with the subject line "Clinical Trials for Pediatric IgAN" at by Friday, February 11, 2022.

Please include the following information with your email:

  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)