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Study Design: Interventional
Conditions: Fatty Liver, Liver Diseases
Division: DDN
Duration: 2012 – Present
# Recruitment Centers: 10
Treatment: Cysteamine
Available Genotype Data: No
Image Summary: No
Transplant Type: None
Does it have dialysis patients: No
Clinical Trials URL:
http://www.clinicaltrials.gov/show/NCT01529268
Data Package Version Number: 2 (Updated on: October 12, 2020)
DOI: 10.58020/637v-9r95
How to cite this dataset: Tonascia, James (2023). Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease (NAFLD) in Children (V2) [Dataset]. NIDDK Central Repository. https://doi.org/10.58020/637v-9r95
Data availability statement: Data from the Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease (NAFLD) in Children [(V2)/https://doi.org/10.58020/637v-9r95] reported here are available for request at the NIDDK Central Repository (NIDDK-CR) website, Resources for Research (R4R), https://repository.niddk.nih.gov/.
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.
The CyNCh trial aims to determine whether treatment with delayed-release cysteamine bitartrate capsules results in improvement in NAFLD severity in children.
The primary outcome measure is improvement in NAFLD at 52 weeks, defined as a decrease in NAFLD Activity Score (NAS) of at least 2 and no worsening of fibrosis.
Secondary outcome measures, also to be assessed at 52 weeks, include reduction in serum aminotransferase and gamma-glytamyl transpeptidase, reduction in MRI-determined hepatic fat fraction, changes in markers of oxidation and anti-oxidant status (malondialdehyde, F2alpha-isoprostane, total antioxidant capacity, oxidized LDL), changes in histology and symptoms, and quality of life.
Children between the ages of 8 to 17 years who met the following criteria were eligible for enrollment:
Exclusion criteria are documented in the study protocol.
This study is ongoing.
Specimens and Study Datasets
| Serum | 12924 |
| Plasma | 6593 |
| Liver Tissue | 3 |
 |
CyNCh_Data_Dictionary (PDF) - 995.5 KB | |
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cynch_dsic (PDF) - 289.8 KB | |
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CyNCh_protocol (PDF) - 463.6 KB | |
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CyNCh_Roadmap_V2 (PDF) - 185.0 KB | |
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Forms (PDF) - 3.2 MB | |
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Standard_Operating_Procedures (PDF) - 16.9 MB |
If you need accessible versions of documents, please email your request to NIDDK-CRsupport@niddk.nih.gov.
