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NIH: National Institute of Diabetes and Digestive and Kidney Diseases
NIDDK Central Repository
Citation
Tonascia, James (2020). Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease (NAFLD) in Children (CyNCh) (Version 2) [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 (CyNCh) [(Version 2) 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/.
Acknowledgement Statement
The CyNCh study was conducted by the study investigators and supported by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The resources from the CyNCh (https://doi.org/10.58020/637v-9r95) study reported here were supplied by NIDDK Central Repository (NIDDK-CR) and are available for request at https://repository.niddk.nih.gov. This manuscript was not prepared under the auspices of the CyNCh study and does not necessarily reflect the opinions or views of the CyNCh study, NIDDK-CR, or NIDDK.
Data Package Version
Version 2 (Updated on: Oct 12, 2020)
Resource Availability
  • Data Available for Request
  • Specimens Available for Request
Publications
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General Description

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.

Objectives

The CyNCh trial aims to determine whether treatment with delayed-release cysteamine bitartrate capsules results in improvement in NAFLD severity in children.

Outcome Measure

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.

Eligibility Criteria

Children between the ages of 8 to 17 years who met the following criteria were eligible for enrollment:

  • Liver biopsy obtained within 90 days of screening visit and not more than 120 days before randomization
  • Clinical history and liver histology consistent with NAFLD
  • No evidence of any other liver disease by clinical history or histological evaluation
  • A histological severity of NAFLD Activity score ≥ 4

Exclusion criteria are documented in the study protocol.

Outcome

This study is ongoing.

Research Area

Liver Disease

Study Type

Interventional

Study Sites

10

Study Start Date

2012-06

Study End Date

2015-09

Condition

Metabolic Dysfunction-Associated Steatotic Liver Disease, Fatty Liver Disease

Clinical Trials URL

http://www.clinicaltrials.gov/show/NCT01529268

Keywords

Nonalcoholic Fatty Liver Disease (NAFLD), Serum Aminotransferase, Cysteamine, Delayed-Release Cysteamine Bitartrate, Gamma-Glytamyl Transpeptidase, Heptic Fat Fraction, Fibrosis, NAFLD Activity Score (NAS), Liver Biopsy

NIDDK Division

Division of Digestive Diseases and Nutrition

169
Participants
Target Population
Children

Public Documents Table
Document Name
Description
Document Type
File Format
Compliance
Download

Non-Public Documents (13)
Non-Public Documents Table
Document Name
Description
Document Type
File Format
Datasets (32)
Datasets Table
Dataset Name
Description
# of Records
# of Variables
File Format(s)
Drug Dispensing Documentation Dataset
Captures drug dispensing documentation data665sas7bdat (128 KB); csv (38.72 KB)
Adverse Event Report Dataset
Captures adverse event data, including type, intubation status, relationship to study drug, severity, outcome, and date of resolution169sas7bdat (128 KB); csv (12.35 KB)
Table 5 One Rec SAE Analysis Dataset
Contains data used to generate Table 5 (One Rec SAE) in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (128 KB); csv (10.14 KB)
Table 5 One Rec Analysis Dataset
Contains data used to generate Table 5 (One Rec) in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (128 KB); csv (10.14 KB)
Table 5 All SAE Analysis Dataset
Contains data used to generate Table 5 (All SAE) in the publication by Schwimmer et al. (2016) in Gastroenterology10sas7bdat (128 KB); csv (1019 B)
Pediatric Quality of Life: Child Report (Age 8-12) Dataset
Captures data from the Pediatric Quality of Life: Child Report (Age 8-12)157sas7bdat (128 KB); csv (19.09 KB)
Study Drug Dispensing and Return Dataset
Captures study drug dispensing and return data1146sas7bdat (192 KB); csv (128.53 KB)
Follow-Up Medical History Dataset
Captures follow-up medical history data917sas7bdat (512 KB); csv (630.43 KB)
Laboratory Tests Done Only During Screening Dataset
Captures laboratory results from tests done only during screening169sas7bdat (128 KB); csv (21.78 KB)
Laboratory Results - Tests Done at Screening and Follow-Up Visits Dataset
Captures laboratory results from tests done at screening and follow-up visits1068sas7bdat (576 KB); csv (229.18 KB)
Table 1 Analysis Dataset
Contains data used to generate Table 1 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (192 KB); csv (44.83 KB)
Table 5 All Analysis Dataset
Contains data used to generate Table 5 (All) in the publication by Schwimmer et al. (2016) in Gastroenterology358sas7bdat (192 KB); csv (25.38 KB)
Supplementary Figure 1 Analysis Dataset
Contains data used to generate Supplementary Figure 1 in the publication by Schwimmer et al. (2016) in Gastroenterology230sas7bdat (128 KB); csv (7.3 KB)
Focused Physical Examination Dataset
Captures focused physical examination data457sas7bdat (256 KB); csv (62.54 KB)
Supplementary Table 3 Analysis Dataset
Contains data used to generate Supplementary Table 3 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (192 KB); csv (86.37 KB)
Table 2 Analysis Dataset
Contains data used to generate Table 2 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (128 KB); csv (20.36 KB)
Registration Dataset
Captures registration data169sas7bdat (128 KB); csv (20.72 KB)
Pediatric Quality of Life: Teen Report (Age 13-17) Dataset
Captures data from the Pediatric Quality of Life: Teen Report (Age 13-17)312sas7bdat (128 KB); csv (37.82 KB)
Baseline History Dataset
Captures baseline history data169sas7bdat (320 KB); csv (167.27 KB)
Supplementary Table 2 Analysis Dataset
Contains data used to generate Supplementary Table 2 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (128 KB); csv (18 KB)
Treatment Dataset
Captures treatment data169sas7bdat (128 KB); csv (2.49 KB)
Genetic Consent and Blood Collection Documentation Dataset
Captures genetic consent and blood collection documentation data at screening visit169sas7bdat (128 KB); csv (5.69 KB)
Table 4 Analysis Dataset
Contains data used to generate Table 4 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (128 KB); csv (20.16 KB)
Physical Examination Dataset
Captures physical exam data, including any abnormal findings616sas7bdat (576 KB); csv (123.69 KB)
Figure 1 Analysis Dataset
Contains data used to generate Figure 1 in the publication by Schwimmer et al. (2016) in Gastroenterology; data includes baseline demographic characteristics, quality of life, laboratory values, inflammation score, steatosis score, hypertension, NAFLD activity score, fibrosis stage, steatohepatitis, and treatment1078sas7bdat (192 KB); csv (86.8 KB)
Central Histology Review Dataset
Captures central histology review data315sas7bdat (128 KB); csv (49.2 KB)
Nutrition Data Documentation Dataset
Captures nutrition data311sas7bdat (128 KB); csv (38.55 KB)
Symptoms of Liver Disease Dataset
Captures symptoms of liver disease data922csv (127.97 KB); sas7bdat (192 KB)
Supplementary Table 1 Analysis Dataset
Contains data used to generate Supplementary Table 1 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (128 KB); csv (6.07 KB)
Pediatric Quality of Life: Parent Report for Children (Age 8-12) Dataset
Captures data from the Pediatric Quality of Life: Parent Report for Children (Age 8-12)157sas7bdat (128 KB); csv (19.09 KB)
Pediatric Quality of Life: Parent Report for Teens (Age 13-17) Dataset
Captures data from the Pediatric Quality of Life: Parent Report for Teens (Age 13-17) 304sas7bdat (128 KB); csv (36.86 KB)
Table 3 Analysis Dataset
Contains data used to generate Table 3 in the publication by Schwimmer et al. (2016) in Gastroenterology169sas7bdat (192 KB); csv (87.52 KB)
Specimens (19,520)
Specimens Table
Specimen
Count
Liver Tissue3
Plasma6593
Serum12924