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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:
- 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.
This study is ongoing.
Liver Disease
Interventional
10
2012-06
2015-09
Metabolic Dysfunction-Associated Steatotic Liver Disease, Fatty Liver Disease
Fibrosis, Gamma-Glytamyl Transpeptidase, Serum Aminotransferase, Nonalcoholic Fatty Liver Disease (NAFLD), Delayed-Release Cysteamine Bitartrate, NAFLD Activity Score (NAS), Heptic Fat Fraction, Cysteamine, Liver Biopsy
DDN
Document Name | Description | Document Type | File Format |
|---|---|---|---|
Dataset Name | Description | # of Records | # of Variables | File Format(s) |
|---|---|---|---|---|
Drug Dispensing Documentation Dataset | Captures drug dispensing documentation data | sas7bdat (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 resolution | sas7bdat (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 Gastroenterology | sas7bdat (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 Gastroenterology | sas7bdat (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 Gastroenterology | sas7bdat (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) | sas7bdat (128 KB); csv (19.09 KB) | ||
Study Drug Dispensing and Return Dataset | Captures study drug dispensing and return data | sas7bdat (192 KB); csv (128.53 KB) | ||
Follow-Up Medical History Dataset | Captures follow-up medical history data | sas7bdat (512 KB); csv (630.43 KB) | ||
Laboratory Tests Done Only During Screening Dataset | Captures laboratory results from tests done only during screening | sas7bdat (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 visits | sas7bdat (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 Gastroenterology | sas7bdat (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 Gastroenterology | sas7bdat (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 Gastroenterology | sas7bdat (128 KB); csv (7.3 KB) | ||
Focused Physical Examination Dataset | Captures focused physical examination data | sas7bdat (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 Gastroenterology | sas7bdat (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 Gastroenterology | sas7bdat (128 KB); csv (20.36 KB) | ||
Registration Dataset | Captures registration data | sas7bdat (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) | sas7bdat (128 KB); csv (37.82 KB) | ||
Baseline History Dataset | Captures baseline history data | sas7bdat (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 Gastroenterology | sas7bdat (128 KB); csv (18 KB) | ||
Treatment Dataset | Captures treatment data | sas7bdat (128 KB); csv (2.49 KB) | ||
Genetic Consent and Blood Collection Documentation Dataset | Captures genetic consent and blood collection documentation data at screening visit | sas7bdat (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 Gastroenterology | sas7bdat (128 KB); csv (20.16 KB) | ||
Physical Examination Dataset | Captures physical exam data, including any abnormal findings | sas7bdat (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 treatment | sas7bdat (192 KB); csv (86.8 KB) | ||
Central Histology Review Dataset | Captures central histology review data | sas7bdat (128 KB); csv (49.2 KB) | ||
Nutrition Data Documentation Dataset | Captures nutrition data | sas7bdat (128 KB); csv (38.55 KB) | ||
Symptoms of Liver Disease Dataset | Captures symptoms of liver disease data | csv (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 Gastroenterology | sas7bdat (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) | sas7bdat (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) | sas7bdat (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 Gastroenterology | sas7bdat (192 KB); csv (87.52 KB) |
Specimen | Count |
|---|---|
| Liver Tissue | 3 |
| Plasma | 6593 |
| Serum | 12924 |