MATCH cohort provides regulatory-ready external control framework for MSUD gene therapy trials
By
Kevin A. Strauss1,2,3 Send email to [email protected]
Summary
This article presents the MSUD Age-matched Standard Treatment Cohort (MATCH), a prospective natural history study of 11 infants with classic maple syrup urine disease (MSUD) followed from neonatal diagnosis to liver transplantation. Designed as a regulatory-ready external control for gene therapy trials, MATCH aligns with FDA guidance and ICH E9(R1) standards, applying prespecified eligibility criteria, fixed visit cadence, adjudicated outcomes, and explicit handling of intercurrent events. The authors demonstrate through Monte Carlo simulations how protocolized natural history data can serve as quantitative evidence supporting ethically defensible single-arm trials in rare diseases where randomized controlled trials are infeasible.
Source
bskyMATCH cohort provides regulatory-ready external control framework for MSUD gene therapy trialscell.comKey quotes
· 3 pulledMaple syrup urine disease (MSUD) is a life-threatening metabolic disorder for which randomized trials are infeasible.
MATCH applies prespecified eligibility criteria, fixed visit cadence, adjudicated outcomes, and explicit handling of intercurrent events.
The authors demonstrate how protocolized data can serve as quantitative evidence supporting ethically defensible single-arm trials.
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