Precision Medicine Proof of Concept for TNF Inhibition in FSGS and Treatment Resistant Minimal Change Disease

Date Added
April 5th, 2022
PRO Number
Pro00117119
Researcher
David Selewski

List of Studies


Keywords
Kidney, Pediatrics
Summary

This research is studying the use of a drug called Humira in a small number of patients with Focal Segmental Glomerulosclerosis (FSGS) or treatment resistant Minimal Change Disease (TR-MCD). Specifically, some patients with FSGS and MCD have been shown in research studies to have overactive tumor necrosis factor (TNF) mechanisms in their kidney tissue. In these patients, high levels of urine markers called MCP1 and TIMP1 have been seen.

In this study, patients with FSGS or MCD with high levels of MCP1 and TIMP1 will be given a medication to block TNF, called Humira, to see if it reduces the urine levels of MCP1 and TIMP1. Subjects' health-related information, blood, and urine will be collected for this research study. This study may help us to develop individualized treatment options for future patients with TNF-driven FSGS or MCD.

Some of the more common risks with taking Humira include: infections, injection site reactions, headache and rash. This study may offer some benefit to subjects now by possibly reducing the amount of protein in their urine. The study may also benefit other patients in the future based on the knowledge gained. Alternatives to joining this study include treatment with steroids or other immunosuppressive medications.

Institution
MUSC
Recruitment Contact
Christian Conley
843-792-1213
conleyc@musc.edu

NEPTUNE

Date Added
February 22nd, 2022
PRO Number
Pro00115978
Researcher
David Selewski

List of Studies


Keywords
Children's Health, Kidney, Pediatrics
Summary

A prospective parallel cohort study generating two groups of participants will be performed in NEPTUNE. The two groups are: (1) Cohort A which includes the FSGS/MCD Cohort; and the MN Cohort, both incipient and prevalent biopsied patients; and (2) Cohort B – a non-biopsy, treatment-naïve, pediatric cohort less than 19 years of age, cNEPTUNE. The sample size for the combined FSGS/MCD and MN Cohorts is a minimum of 800 participants, with a minimum of 375 new patients recruited under Protocol V5.0. The sample size for the second group, cNEPTUNE, will be a minimum of 200 participants. Participants will be recruited into each subgroup concurrently. All participants who meet the inclusion criteria at the participating centers will be enrolled if the participants or their legally authorized representative(s) provide comprehensive written informed consent. A recruit-to-replace strategy will be employed throughout the enrollment phase. Cohort A study visits including screening/eligibility, baseline, biopsy, and follow-up visits, and SMS texting. Study visits for Cohort B, cNEPTUNE, including screening/eligibility, baseline, follow-up visits, and SMS texting.

Institution
MUSC
Recruitment Contact
Christian Conley
843-792-1213
conleyc@musc.edu



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