Clinical Trials Office

Research Study for Children with Non-Alcoholic Fatty Liver Disease (NAFLD)

What is the purpose of this study?

Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to evaluate whether treatment with an investigational medication, cysteamine bitartrate delayed release (DR), will help improve the livers of children diagnosed with Nonalcoholic Fatty Liver Disease (NAFLD).

Who can participate?

Children and teens 8 to 17 years of age with NAFLD will be eligible to participate. Participants must have a baseline biopsy confirming a diagnosis of NAFLD and no history of poorly managed diabetes, heart disease or other chronic liver diseases.

What is involved?

What is involved?

Participants will be randomly chosen to receive either the study drug (cysteamine bitartrate DR) or a placebo (a substance that looks like the study drug but contains no medication).

The treatment part of the study lasts for approximately one year and post-treatment follow-up lasts for approximately 24-weeks. 

The following is a list of some of the procedures that will take place during the study (in addition to regular clinic visits):

  • Review of medical history and current medications
  • Check of vital signs (blood pressure, heart rate, respiratory rate, body temperature)
  • Standardized lifestyle interventions (for example, strategies to reduce saturated fat and increase physical activity)
  • Blood draws
  • Magnetic Resonance Imaging (MRI) scan
  • End-of-treatment liver biopsy 

What are the benefits?

Your child might not receive a direct medical benefit.  However, the information gained from this study may benefit individuals with NAFLD in the future.

Will I get all the facts about the study?

Parents interested in having their child participate will be given a consent form that thoroughly explains all of the details of the study. The form covers all of the procedures, the risks, the benefits, the pay, who to contact with questions or concerns and more. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered. Study procedures will not begin until a parent or guardian has signed this form and, if of age, the child has given at minimum his/her verbal agreement.

Do participants receive pay, compensation or reimbursement?

Participants will be paid up to $450, in the form of gift cards, for completion of all study visits and procedures. 

Who should I contact for more information?

Kimberlee Bernstein

kimberlee.bernstein@cchmc.org

513-636-4406

Steatohepatitis Center

Division of Gastroenterology, Hepatology and Nutrition

Cincinnati Children’s Hospital Medical Center, MLC 2010

3333 Burnet Ave.

Cincinnati, OH  45229-3039

Principal Investigator

Stavra Xanthakos, MD, MS

Steatohepatitis Center

Division of Gastroenterology, Hepatology and Nutrition

Cincinnati Children’s Hospital Medical Center

Co-Investigator:

Rohit Kohli, MBBS, MS

Steatohepatitis Center

Division of Gastroenterology, Hepatology and Nutrition

Cincinnati Children’s Hospital Medical Center