MAS825 Trial for NLRC4-GOF

MAS825 Trial for NLRC4-GOF

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 If you are interested in participating in the study or want to learn more, please contact: Contact us
This study is now recruiting. Learn more about enrolling here.

Description

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

Eligibility and criteria
IRB Number:
21-018760
Clinical trial phase:
Phase II
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

What to expect
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.

Related specialties
Smiling CHOP patient holding shopping bag

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