Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease (NAFLD) in Children

The CyNCh (Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children) study is a prospective, multicenter, double-blind clinical trial designed by the NASH Clinical Research Network to determine whether treatment with cysteamine improves disease severity in children diagnosed with NAFLD. Children between the ages of 8 and 17 years with biopsy-confirmed moderate to severe NAFLD were eligible for the CyNCh study. Participants were enrolled and randomized to treatment with either delayed-release cysteamine bitartrate capsules or placebo capsules. Improvement in NAFLD at 52 weeks, defined as a decrease in NAFLD Activity Score (NAS) of at least 2 and no worsening of fibrosis, was assessed as the primary outcome measure. Secondary outcome measures, assessed at 52 weeks, included reduction in serum aminotransferase and gamma-glytamyl transpeptidase, reduction in MRI-determined hepatic fat fraction, changes in markers of oxidation and anti-oxidant status, changes in histology and symptoms, and self-reported quality of life.