Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Nonalcoholic Fatty Liver Disease (NAFLD)

In brief

CyNCh is a multi-center, placebo-controlled clinical trial of children ages 8 to 17 years with biopsy-confirmed moderate to severe nonalcoholic fatty liver disease (NAFLD). The primary objective is to evaluate whether 52 weeks of treatment with cysteamine bitartrate delayed-release capsules will result in improvement in liver disease severity.

Key facts

Study ID

NCT01529268

Run by

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

People needed

169

Starts

2012-06-01

Expected to finish

2015-09-01

Last updated by the study team

2021-06-10

Who can join

Age: 8 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Children age 8-17 years
• Liver biopsy obtained within 90 days of screening visit and not more than 120 days before randomization
• Clinical history consistent with nonalcoholic fatty liver disease (NAFLD)
• Definite NAFLD based upon liver histology
• No evidence of any other liver disease by clinical history or histological evaluation
• A histological severity of: NAFLD Activity Score (NAS) ≥ 4.
• Sexually active female participants of childbearing potential (i.e., not surgically sterile [defined as tubal ligation, hysterectomy, or bilateral oophorectomy]) must agree to utilize the same two acceptable forms of contraception from screening through completion of the study and to complete a serum pregnancy test at each study visit. The acceptable forms of contraception for this study include hormonal contraceptives (oral, implant, transdermal patch, or injection) at a stable dose for at least 3 months prior to screening, and barrier (condom with spermicide, diaphragm with spermicide). Sexual activity will be ascertained at each study visit for post-menarchal females and if sexually active, subject must verify use of the same 2 acceptable forms of contraception. For pre-pubescent children, a documented attestation of abstinence from their parent or guardian will be acceptable.
• Participants must be able to swallow DR Cysteamine tablets with the tablet intact
• Written informed consent from parent or legal guardian
• Written informed assent from the child

You may not qualify if…

• There will be no exclusion criteria based on race, ethnicity or gender.
• Participants with a current history of the following conditions or any other health issues that make it unsafe for them to participate in the opinion of the Investigators:
• Inflammatory bowel disease (if currently active) or prior resection of small intestine
• Heart disease (e.g., myocardial infarction, heart failure, unstable arrhythmias)
• Seizure disorder
• Active coagulopathy
• Gastrointestinal ulcers/bleeding
• Renal dysfunction with a creatinine clearance < 90 mL/min/m2
• History of active malignant disease requiring chemotherapy within the past 12 months prior to randomization
• History of significant alcohol intake (AUDIT questionnaire) or inability to quantify alcohol consumption
• Chronic use (more than 2 consecutive weeks) of medications known to cause hepatic steatosis or steatohepatitis (systemic glucocorticoids, tetracycline, anabolic steroids, valproic acid, salicylates, tamoxifen) in the past year.
• The use of other known hepatotoxins within 90 days of liver biopsy or within 120 days of randomization
• Initiation of medications with the intent to treat NAFLD/NASH in the time period following liver biopsy and prior to randomization
• History of total parenteral nutrition (TPN) use in year prior to screening
• History of bariatric surgery or planning to undergo bariatric surgery during study duration
• Clinically significant depression (patients hospitalized for suicidal ideations or suicide attempts within the past 12 months)
• Any female nursing, planning a pregnancy, known or suspected to be pregnant, or who has a positive serum pregnancy screen.
• Non-compensated liver disease with any one of the following hematologic, biochemical, and serological criteria on entry into protocol:
• Hemoglobin < 10 g/dL;
• White blood cell (WBC) < 3,500 cells/mm3 of blood;
• Neutrophil count < 1,500 cells/mm3 of blood;
• Platelets < 130,000 cells/mm3 of blood;
• Direct bilirubin > 1.0 mg/dL
• Total bilirubin >3 mg/dL
• Albumin < 3.2 g/dL

Where it is running

• University of California, San Diego — San Diego, California, United States
• University of California, San Francisco — San Francisco, California, United States
• Emory University — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago (NWU) — Chicago, Illinois, United States
• Indiana University — Indianapolis, Indiana, United States
• St. Louis University — St Louis, Missouri, United States
• Columbia University — New York, New York, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Texas Children's Hospital — Houston, Texas, United States
• University of Washington, Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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