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

This study has been completed.
Sponsor:
Collaborator:
Raptor Pharmaceuticals Corp.
Information provided by (Responsible Party):
Joel Lavine, University of California, San Diego
ClinicalTrials.gov Identifier:
NCT00799578
First received: November 28, 2008
Last updated: December 13, 2013
Last verified: December 2013
  Purpose

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.

  Show Detailed Description

  Eligibility

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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00799578

Locations
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:
Publications:

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
Cysteamine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 16, 2014