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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
Metabolic Dysfunction-Associated Steatotic Liver Disease, Fatty Liver Disease
Cysteamine
None
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
Division of Digestive Diseases and Nutrition (DDN)
Use is allowed only for the specified disease(s), disorder(s), condition(s), or research area(s): Type 1 Diabetes Mellitus
This NIDDK-funded study is covered by a Certificate of Confidentiality. More information on what this means to Requestors is available in the NIH FAQ.
Dataset Name | Description | # of Records | # of Variables | File Format(s) |
|---|---|---|---|---|
Drug Dispensing Documentation Dataset | Captures drug dispensing documentation data | 665 | 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 | 169 | 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 | 169 | 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 | 169 | 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 | 10 | 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) | 157 | sas7bdat (128 KB); csv (19.09 KB) | |
Study Drug Dispensing and Return Dataset | Captures study drug dispensing and return data | 1146 | sas7bdat (192 KB); csv (128.53 KB) | |
Follow-Up Medical History Dataset | Captures follow-up medical history data | 917 | sas7bdat (512 KB); csv (630.43 KB) | |
Laboratory Tests Done Only During Screening Dataset | Captures laboratory results from tests done only during screening | 169 | 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 | 1068 | 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 | 169 | 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 | 358 | 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 | 230 | sas7bdat (128 KB); csv (7.3 KB) | |
Focused Physical Examination Dataset | Captures focused physical examination data | 457 | 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 | 169 | 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 | 169 | sas7bdat (128 KB); csv (20.36 KB) | |
Registration Dataset | Captures registration data | 169 | 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) | 312 | sas7bdat (128 KB); csv (37.82 KB) | |
Baseline History Dataset | Captures baseline history data | 169 | 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 | 169 | sas7bdat (128 KB); csv (18 KB) | |
Treatment Dataset | Captures treatment data | 169 | 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 | 169 | 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 | 169 | sas7bdat (128 KB); csv (20.16 KB) | |
Physical Examination Dataset | Captures physical exam data, including any abnormal findings | 616 | 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 | 1078 | sas7bdat (192 KB); csv (86.8 KB) | |
Central Histology Review Dataset | Captures central histology review data | 315 | sas7bdat (128 KB); csv (49.2 KB) | |
Nutrition Data Documentation Dataset | Captures nutrition data | 311 | sas7bdat (128 KB); csv (38.55 KB) | |
Symptoms of Liver Disease Dataset | Captures symptoms of liver disease data | 922 | 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 | 169 | 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) | 157 | 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) | 304 | 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 | 169 | sas7bdat (192 KB); csv (87.52 KB) |
Specimen | Count |
|---|---|
| Liver Tissue | 3 |
| Plasma | 6776 |
| Serum | 12932 |