MAS825 Trial for NLRC4-GOF

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The purpose of this study is to find out if the study drug, MAS825, is effective in preventing flares and safe for children with NLRC4-Gain of Function (GOF). Researchers will also be testing how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body (pharmacokinetics). Subjects will be asked to come into the Rheumatology clinic for blood and stool testing, physical exam, an electrocardiogram (ECG), questionnaire completion, and administration of study drug. Participation will last for up to 4 years.

Eligibility & Criteria

IRB #:
21-018760
Official Title:
A Three-Period Multicenter Study, with a Randomized-Withdrawal, Double-Blinded, Placebo-Controlled Design in Period 2 to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in NLRC4-GOF Patients
Study Phase:
Phase II
Eligible Age Range:
0 Months - 18 Years
Gender:
All
Study Categories:

Visit Criteria

The study has three periods:
  • Period 1 subjects will receive study drug at CHOP every 2 weeks for up to 28 weeks
  • Period 2 subjects will be randomly assigned to receive either the study drug or a placebo at CHOP every 2 weeks for up to 24 weeks
  • Period 3 subjects will receive study drug every 2 weeks for up to 3 years.
Leaders
Edward M. Behrens

Edward M. Behrens, MD

Scott W. Canna, MD

Scott W. Canna, MD