NCT00799578

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Oct 2008

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2008

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

November 28, 2008

Completed
3 days until next milestone

First Posted

Study publicly available on registry

December 1, 2008

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2009

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2010

Completed
4.1 years until next milestone

Results Posted

Study results publicly available

January 31, 2014

Completed
Last Updated

January 31, 2014

Status Verified

December 1, 2013

Enrollment Period

8 months

First QC Date

November 28, 2008

Results QC Date

December 13, 2013

Last Update Submit

December 13, 2013

Conditions

Fatty Liver

Keywords

NASH, NAFLD,nonalcoholic steatohepatitis

Outcome Measures

Primary Outcomes (1)

  • Normalization or >50% of Serum ALT Levels From Baseline

    6 months

Study Arms (1)

Cystagon-EC

EXPERIMENTAL
Drug: Cysteamine

Interventions

CysteamineDRUG

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.

Cystagon-EC

Eligibility Criteria

Age10 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of California, San Diego School of Medicine General Clinic Research Center

San Diego, California, 92103-8203, United States

Location

Related Publications (16)

  • Skelly AH, Arcury TA, Gesler WM, Cravey AJ, Dougherty MC, Washburn SA, Nash S. Sociospatial knowledge networks: appraising community as place. Res Nurs Health. 2002 Apr;25(2):159-70. doi: 10.1002/nur.10024.

    PMID: 11933009BACKGROUND

  • Angulo P, Lindor KD. Non-alcoholic fatty liver disease. J Gastroenterol Hepatol. 2002 Feb;17 Suppl:S186-90. doi: 10.1046/j.1440-1746.17.s1.10.x.

    PMID: 12000605BACKGROUND

  • Contos MJ, Sanyal AJ. The clinicopathologic spectrum and management of nonalcoholic fatty liver disease. Adv Anat Pathol. 2002 Jan;9(1):37-51. doi: 10.1097/00125480-200201000-00005.

    PMID: 11756758BACKGROUND

  • Fishbein MH, Miner M, Mogren C, Chalekson J. The spectrum of fatty liver in obese children and the relationship of serum aminotransferases to severity of steatosis. J Pediatr Gastroenterol Nutr. 2003 Jan;36(1):54-61. doi: 10.1097/00005176-200301000-00012.

    PMID: 12499997BACKGROUND

  • Wu G, Fang YZ, Yang S, Lupton JR, Turner ND. Glutathione metabolism and its implications for health. J Nutr. 2004 Mar;134(3):489-92. doi: 10.1093/jn/134.3.489.

    PMID: 14988435BACKGROUND

  • Prescott LF, Critchley JA. The treatment of acetaminophen poisoning. Annu Rev Pharmacol Toxicol. 1983;23:87-101. doi: 10.1146/annurev.pa.23.040183.000511.

    PMID: 6347057BACKGROUND

  • Prescott LF, Park J, Proudfoot AT. Cysteamine, L-methionine and D-penicillamine in paracetamol poisoning. J Int Med Res. 1976;4(4 Suppl):112-7. doi: 10.1177/14732300760040S420. No abstract available.

    PMID: 1026552BACKGROUND

  • Prescott LF. Haemodialysis in paracetomol self-poisoning. Lancet. 1972 Sep 23;2(7778):652. doi: 10.1016/s0140-6736(72)93038-3. No abstract available.

    PMID: 4116795BACKGROUND

  • Prescott LF, Stewart MJ, Proudfoot AT. Cysteamine or N-acetylcysteine for paracetamol poisoning? Br Med J. 1978 Apr 1;1(6116):856-7. doi: 10.1136/bmj.1.6116.856-a. No abstract available.

    PMID: 638489BACKGROUND

  • Mitchell JR, Thorgeirsson SS, Potter WZ, Jollow DJ, Keiser H. Acetaminophen-induced hepatic injury: protective role of glutathione in man and rationale for therapy. Clin Pharmacol Ther. 1974 Oct;16(4):676-84. doi: 10.1002/cpt1974164676. No abstract available.

    PMID: 4417718BACKGROUND

  • Lyons J, Rauh-Pfeiffer A, Yu YM, Lu XM, Zurakowski D, Tompkins RG, Ajami AM, Young VR, Castillo L. Blood glutathione synthesis rates in healthy adults receiving a sulfur amino acid-free diet. Proc Natl Acad Sci U S A. 2000 May 9;97(10):5071-6. doi: 10.1073/pnas.090083297.

    PMID: 10792033BACKGROUND

  • Lu SC. Regulation of glutathione synthesis. Curr Top Cell Regul. 2000;36:95-116. doi: 10.1016/s0070-2137(01)80004-2. No abstract available.

    PMID: 10842748BACKGROUND

  • Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002 Jul 11;347(2):111-21. doi: 10.1056/NEJMra020552. No abstract available.

    PMID: 12110740BACKGROUND

  • Dohil R, Fidler M, Barshop BA, Gangoiti J, Deutsch R, Martin M, Schneider JA. Understanding intestinal cysteamine bitartrate absorption. J Pediatr. 2006 Jun;148(6):764-9. doi: 10.1016/j.jpeds.2006.01.050.

    PMID: 16769383BACKGROUND

  • Pamuk GE, Sonsuz A. N-acetylcysteine in the treatment of non-alcoholic steatohepatitis. J Gastroenterol Hepatol. 2003 Oct;18(10):1220-1. doi: 10.1046/j.1440-1746.2003.03156.x. No abstract available.

    PMID: 12974918BACKGROUND

  • Dohil R, Schmeltzer S, Cabrera BL, Wang T, Durelle J, Duke KB, Schwimmer JB, Lavine JE. Enteric-coated cysteamine for the treatment of paediatric non-alcoholic fatty liver disease. Aliment Pharmacol Ther. 2011 May;33(9):1036-44. doi: 10.1111/j.1365-2036.2011.04626.x. Epub 2011 Mar 13.

    PMID: 21395631DERIVED

Related Links

MeSH Terms

Conditions

Fatty LiverNon-alcoholic Fatty Liver Disease

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System Diseases

Intervention Hierarchy (Ancestors)

MercaptoethylaminesEthylaminesAminesOrganic ChemicalsSulfhydryl CompoundsSulfur Compounds

Results Point of Contact

Title
Dr. Joel Lavine
Organization
Columbia University
jl3553@columbia.edu
212 304 5533

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

November 28, 2008

First Posted

December 1, 2008

Study Start

October 1, 2008

Primary Completion

June 1, 2009

Study Completion

January 1, 2010

Last Updated

January 31, 2014

Results First Posted

January 31, 2014

Record last verified: 2013-12

Locations

US(1)