Trial · NCT01529268
Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children
NCT01529268, Phase 2, Phase 3, Completed, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); results first posted 2017-09-05
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ConductScience Library. Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children. Trials, record NCT01529268. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01529268
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children
- NCT number
- NCT01529268
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 169 participants, actual
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Conditions
- Nonalcoholic Fatty Liver Disease (NAFLD)
- Interventions
- DR cysteamine bitartrate capsule, DR cysteamine bitartrate placebo
- First posted
- 2012-02-08
- Last updated
- 2021-06-10
- Results first posted
- 2017-09-05
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) | 52 weeks | Centrally scored and masked assessment of histologic improvement in Nonalcholic Fatty Liver Disease (NAFLD) between the baseline liver biopsy and follow-up biopsy after 52 weeks of treatment, where improvement is defined as: (1) decrease in the NAFLD Activity Score (NAS) of 2 or more and (2) no worsening of fibrosis. |
| Secondary | Change in Nonalcoholic Fatty Liver Disease (NAFLD) Activity Score (NAS) | 52 weeks | Change from baseline in the NAFLD Activity Score (NAS), which is a composite score equal to the sum of the steatosis grade (0-3), lobular inflammation grade (0-3), and hepatocellular ballooning grade (0-2), from centralized pathologist scoring of liver biopsies. The overall scale of the NAS is 0-8, with higher scores indicating more severe disease. The outcome measure, change from baseline in NAFLD Activity Score (NAS), has a possible range from -8 to +8, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome. Components of the NAS are scored as follows: Steatosis grade 0=\<5% steatosis, 1=5-33% steatosis, 2=34-66% steatosis, 3=\>66% steatosis. Lobular inflammation grade=amount of lobular inflammation (combines mononuclear, fat granulomas, and polymorphonuclear (pmn) foci): 0=0, 1=\<2 under 20x magnification, 2=2-4 under 20x magnification, 3=\>4 under 20x magnification. Hepatocellular ballooning 0=none, 1=mild, 2=more than mild. |
| Secondary | Steatosis: Patients With Improvement | 52 weeks | Improvement in steatosis defined as any decrease in steatosis grade comparing 52-week biopsy to baseline. |
| Secondary | Steatosis: Change in Score | 52 weeks | Change from baseline in steatosis score. Steatosis score is based on central pathologist grading of liver biopsies: 0=\<5% steatosis; 1=5-33% steatosis, 2=34-66% steatosis, 3=\>66% steatosis. Change in steatosis score has a possible range of -3 to +3, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement). |
| Secondary | Lobular Inflammation: Patients With Improvement | 52 weeks | Improvement in lobular inflammation defined as any decrease in lobular inflammation grade comparing 52-week biopsy to baseline. |
| Secondary | Lobular Inflammation: Change in Score | 52 weeks | Change from baseline in lobular inflammation score. The amount of lobular inflammation is based on central pathologist grading of liver biopsies, and combines mononuclear, fat granulomas, and polymorphonuclear (pmn) foci: 0=none; 1=\<2 under 20x magnification, 2=2-4 under 20x magnification, 3=\>4 under 20x magnification. Change in lobular inflammation score has a possible range of -3 to +3, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement). |
| Secondary | Hepatocellular Ballooning: Patients With Improvement | 52 weeks | Improvement in hepatocellular ballooning defined as any decrease in hepatocellular ballooning score comparing 52-week biopsy to baseline. |
| Secondary | Hepatocellular Ballooning: Change in Score | 52 weeks | Change from baseline in hepatocellular ballooning score. The amount of hepatocellular ballooning is based on central pathologist grading of liver biopsies: 0=none; 1=few ballooned hepatocytes, 2=many ballooned hepatocytes. Change in hepatocellular ballooning score has a possible range of -2 to +2, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement). |
| Secondary | Portal Inflammation: Patients With Improvement | 52 weeks | Improvement in portal inflammation defined as any decrease in portal inflammation score comparing 52-week biopsy to baseline. |
| Secondary | Portal Inflammation: Change in Score | 52 weeks | Change from baseline in portal inflammation score. The amount of portal inflammation is based on central pathologist grading of liver biopsies: 0=none; 1=mild, 2=more than mild. Change in portal inflammation score has a possible range of -2 to +2, with negative values indicating a better outcome (improvement) and positive values indicating a worse outcome (no improvement). |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes25
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) | 52 weeks | Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement. | participants | NUMBER |
| SECONDARY | Change in Nonalcoholic Fatty Liver Disease (NAFLD) Activity Score (NAS) | 52 weeks | The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis). | units on a scale | MEAN |
| SECONDARY | Steatosis: Patients With Improvement | 52 weeks | Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement. | participants | NUMBER |
| SECONDARY | Steatosis: Change in Score | 52 weeks | The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis). | units on a scale | MEAN |
| SECONDARY | Lobular Inflammation: Patients With Improvement | 52 weeks | Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement. | participants | NUMBER |
| SECONDARY | Lobular Inflammation: Change in Score | 52 weeks | The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis). | units on a scale | MEAN |
| SECONDARY | Hepatocellular Ballooning: Patients With Improvement | 52 weeks | Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement. | participants | NUMBER |
| SECONDARY | Hepatocellular Ballooning: Change in Score | 52 weeks | The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis). | units on a scale | MEAN |
| SECONDARY | Portal Inflammation: Patients With Improvement | 52 weeks | Analysis based on intention to treat; patients with missing 52-week biopsy were imputed as lack of improvement. | participants | NUMBER |
| SECONDARY | Portal Inflammation: Change in Score | 52 weeks | The smaller number of participants analyzed is due to missing 52-week biopsies (complete case analysis). | units on a scale | MEAN |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) | DR Cysteamine Bitartrate Capsule | 25 | participants | ||
| Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) | DR Cysteamine Bitartrate Placebo | 18 | participants | ||
| Change in Nonalcoholic Fatty Liver Disease (NAFLD) Activity Score (NAS) | DR Cysteamine Bitartrate Capsule | -0.8 | Standard Deviation 1.8 | units on a scale | |
| Change in Nonalcoholic Fatty Liver Disease (NAFLD) Activity Score (NAS) | DR Cysteamine Bitartrate Placebo | -0.8 | Standard Deviation 1.8 | units on a scale | |
| Steatosis: Patients With Improvement | DR Cysteamine Bitartrate Capsule | 26 | participants | ||
| Steatosis: Patients With Improvement | DR Cysteamine Bitartrate Placebo | 33 | participants | ||
| Steatosis: Change in Score | DR Cysteamine Bitartrate Capsule | -0.3 | Standard Deviation 0.9 | units on a scale | |
| Steatosis: Change in Score | DR Cysteamine Bitartrate Placebo | -0.4 | Standard Deviation 0.9 | units on a scale | |
| Lobular Inflammation: Patients With Improvement | DR Cysteamine Bitartrate Capsule | 32 | participants | ||
| Lobular Inflammation: Patients With Improvement | DR Cysteamine Bitartrate Placebo | 17 | participants |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | DR Cysteamine Bitartrate Capsule | 88 | |
| Overall Study | STARTED | DR Cysteamine Bitartrate Placebo | 81 | |
| Overall Study | Completed 52-week Biopsy | DR Cysteamine Bitartrate Capsule | 71 | |
| Overall Study | Completed 52-week Biopsy | DR Cysteamine Bitartrate Placebo | 75 | |
| Overall Study | COMPLETED | DR Cysteamine Bitartrate Capsule | 76 | |
| Overall Study | COMPLETED | DR Cysteamine Bitartrate Placebo | 78 | |
| Overall Study | NOT COMPLETED | DR Cysteamine Bitartrate Capsule | 12 | |
| Overall Study | NOT COMPLETED | DR Cysteamine Bitartrate Placebo | 3 |
Source ClinicalTrials.gov
Adverse events218
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pain - abdomen NOS | Gastrointestinal disorders | DR Cysteamine Bitartrate Capsule | 11 | 88 | 11 |
| other | Pain - abdomen NOS | Gastrointestinal disorders | DR Cysteamine Bitartrate Placebo | 8 | 81 | 9 |
| other | Ocular - other | Eye disorders | DR Cysteamine Bitartrate Capsule | 1 | 88 | 1 |
| other | Ocular - other | Eye disorders | DR Cysteamine Bitartrate Placebo | 0 | 81 | 0 |
| other | Allergic reaction | Immune system disorders | DR Cysteamine Bitartrate Capsule | 1 | 88 | 1 |
| other | Allergic reaction | Immune system disorders | DR Cysteamine Bitartrate Placebo | 1 | 81 | 1 |
| other | Rhinitis | Immune system disorders | DR Cysteamine Bitartrate Capsule | 3 | 88 | 3 |
| other | Rhinitis | Immune system disorders | DR Cysteamine Bitartrate Placebo | 5 | 81 | 5 |
| other | Allergy - other | Immune system disorders | DR Cysteamine Bitartrate Capsule | 0 | 88 | 0 |
| other | Allergy - other | Immune system disorders | DR Cysteamine Bitartrate Placebo | 1 | 81 | 1 |
1 to 10 of 218Source ClinicalTrials.gov
See all 218 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DR Cysteamine Bitartrate Capsule | 0 | 88 |
| Total, all-cause mortality | DR Cysteamine Bitartrate Placebo | 0 | 81 |
| Total, other adverse events | DR Cysteamine Bitartrate Capsule | 62 | 88 |
| Total, other adverse events | DR Cysteamine Bitartrate Placebo | 55 | 81 |
| Total, serious adverse events | DR Cysteamine Bitartrate Capsule | 5 | 88 |
| Total, serious adverse events | DR Cysteamine Bitartrate Placebo | 4 | 81 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants9
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|---|---|---|---|---|---|
| U01DK061713 | Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) | DIEHL, ANNA MAE ELIZABETH | DUKE UNIVERSITY | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061718 | The Saint Louis University Component of the NASH Clinical Research Network | NEUSCHWANDER-TETRI, BRENT A | SAINT LOUIS UNIVERSITY | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061728 | Clinical Research Network in Nonalcoholic Steatohepatitis | KOWDLEY, KRIS | LIVER INSTITUTE NORTHWEST PLLC | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061731 | Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) | SANYAL, ARUN J | VIRGINIA COMMONWEALTH UNIVERSITY | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061732 | Continuation of the Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Clinical Center-Cleveland site | DASARATHY, SRINIVASAN | CLEVELAND CLINIC LERNER COM-CWRU | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061734 | Clinical Research on Nonalcoholic Fatty Liver Disease | LOOMBA, ROHIT | UNIVERSITY OF CALIFORNIA, SAN DIEGO | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061737 | Collaborative Clinical Research in Nonalcoholic Steatohepatitis | CHALASANI, NAGA P | INDIANA UNIVERSITY INDIANAPOLIS | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061738 | Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) | TERRAULT, NORAH A. | UNIVERSITY OF SOUTHERN CALIFORNIA | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2025 | NIH RePORTER |
| U01DK061730 | Continuation of the Nonalcoholic Steatohepatitis Clinical Research Network | TONASCIA, JAMES A | JOHNS HOPKINS UNIVERSITY | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2002 to 2018 | NIH RePORTER |
Trial comparisons9
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Portal Inflammation: Patients With Improvement | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Fibrosis: Patients With Improvement | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Lobular Inflammation: Patients With Improvement | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Steatosis: Patients With Improvement | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Hepatocellular Ballooning: Patients With Improvement | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: Resolution of NASH | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: deaths from any cause | Trial comparisons |
| DR Cysteamine Bitartrate Capsule vs DR Cysteamine Bitartrate Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01529268
GET https://conductscience.org/api/trials/v1/NCT01529268/registered?page=2