A Preliminary Study to Evaluate Cysteamine Therapy in Human Subjects With Non-Alcoholic Steatohepatitis (NASH)

This study has been completed.
Raptor Pharmaceuticals Corp.
Information provided by (Responsible Party):
Joel Lavine, University of California, San Diego
ClinicalTrials.gov Identifier:
First received: November 28, 2008
Last updated: December 13, 2013
Last verified: December 2013

The purpose of this study is to determine whether cysteamine will effectively reduce or reverse the biologic markers of steatohepatitis in patients.

Condition Intervention Phase
Fatty Liver
Drug: Cysteamine
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by University of California, San Diego:

Primary Outcome Measures:
  • Normalization or >50% of Serum ALT Levels From Baseline [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Enrollment: 13
Study Start Date: October 2008
Study Completion Date: January 2010
Primary Completion Date: June 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cystagon-EC Drug: Cysteamine
Drug is in enteric-coated capsule form. The dosage will begin at 1g/m-squared body surface area with a maximum dose of 1000mg twice daily. Treatment period is 3-6 months.

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Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Biopsy confirmed diagnosis of non-alcoholic steatohepatitis (within past 12 months)
  • Ages 10 yrs and older
  • Must swallow tablets on a regular basis
  • ALT level >60 iu/L

Exclusion Criteria:

  • Subjects with known hypersensitivity to cysteamine
  • History, currently or within the past 3 months, of the following conditions:
  • Pancreatitis
  • Inflammatory bowel disease
  • Malabsorption
  • Unstable heart disease, e.g., myocardial infarction, heart failure, arrhythmias.
  • Unstable diabetes mellitus
  • Any bleeding disorder.
  • Zollinger-Ellison syndrome
  • Malignant disease
  • Subjects whom maybe pregnant or have health issues that make it unsafe for them participate, or whose concomitant medical problems preclude them from committing to the study schedule.
  • No specific NASH medical therapy for 3 months such as vitamin E, s-adenosyl methionine or metformin or other NAFLD study drugs.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00799578

United States, California
University of California, San Diego School of Medicine General Clinic Research Center
San Diego, California, United States, 92103-8203
Sponsors and Collaborators
Joel Lavine
Raptor Pharmaceuticals Corp.
  More Information

Additional Information:

Responsible Party: Joel Lavine, Professor, University of California, San Diego
ClinicalTrials.gov Identifier: NCT00799578     History of Changes
Other Study ID Numbers: 07-1699
Study First Received: November 28, 2008
Results First Received: December 13, 2013
Last Updated: December 13, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of California, San Diego:
NASH, NAFLD,nonalcoholic steatohepatitis

Additional relevant MeSH terms:
Fatty Liver
Liver Diseases
Digestive System Diseases
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014