Clinical Trial: TP10 Use in Patients With C3 Glomerulopathy (C3G)

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: A Pilot, Open-Label Single Center Trial of TP10 in Pediatric and Adult Patients With C3 Glomerulopathy (C3G)

Brief Summary: The purpose of this study is to evaluate the safety of repeated TP10 dosing in pediatric and adult patients with C3G and to evaluate the activity of TP10 in pediatric and adult patients with C3G, as measured by the proportion of patients with normalization of serum C3, serum C3 breakdown products, or alternative pathway (AP) complement activity.

Detailed Summary:

Overview of Study Design: This study is a pilot Phase I, open-label, non-randomized, single-arm, clinical trial of TP10 in 5 patients with C3G, aged 4 years or older. The study consists of a 4-week screening period followed by a 26-week treatment period. (See Schedule of Events, Table 1.) The 26-week treatment period is further divided into an Induction Period of up to 4 weeks, followed by a Maintenance Period, which allows for continued treatment to a total of 26 weeks.

Screening Period: Patients will be identified for possible enrollment in this study through a number of mechanisms, including physician or patient-self referral and participation in an on-going study of C3G in which renal pathology is reviewed to confirm the diagnosis of C3G and genetic and complement studies are performed. Patients who appear to meet eligibility criteria for this study upon initial review will be asked to contact Drs. Nester and Smith if they are interested in this study. Those patients who express interest in this study will be scheduled for a screening clinic visit at which time they will meet the study team and review the study design and requirements. A consent for study participation will be obtained. During this visit those events/tests noted on Table 1, Screening Visit, will be completed. If not already vaccinated within the time period of active coverage specified by the vaccine manufacturer, patients will be administered meningococcal, pneumococcal and haemophilus influenzae vaccines prior to study drug administration.

Treatment Period: All patients will be enrolled through the University of Iowa. This study will follow a patient-specific dose-escalation scheme during the Induction Period and subsequent dose adjustments based on complement levels during the Maintenance Period (as described under Investigational Product Dosing and
Sponsor: University of Iowa

Current Primary Outcome: C3 serum measurements, serum C3 breakdown products, and/or alternative pathway (AP) complement activity. [ Time Frame: 2 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Appropriate dose range and regimen for TP10. [ Time Frame: 2 years ]

Original Secondary Outcome: Same as current

Information By: University of Iowa

Dates:
Date Received: November 5, 2014
Date Started: November 2014
Date Completion:
Last Updated: October 25, 2016
Last Verified: October 2016