Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 19 of 77 for:    Long-chain fatty acids

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00004418
Recruitment Status : Suspended (No funding and moved to expanded access)
First Posted : October 19, 1999
Last Update Posted : September 18, 2014
Sponsor:
Information provided by (Responsible Party):
Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Tracking Information
First Submitted Date  ICMJE October 18, 1999
First Posted Date  ICMJE October 19, 1999
Last Update Posted Date September 18, 2014
Study Start Date  ICMJE April 1998
Estimated Primary Completion Date November 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 5, 2013)
neurological disability [ Time Frame: 6 months post intervention, then every 3 months until age 13 years ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00004418 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 5, 2013)
magnetic resonance imaging of the head [ Time Frame: yearly ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: February 5, 2013)
very long chain fatty acids (VLCFA) [ Time Frame: every month during first year, then every 1-3 months ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Official Title  ICMJE Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
Brief Summary

OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.

II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.

Detailed Description

PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.

Patients are followed monthly for 6 months, then every 3 months until death.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Adrenoleukodystrophy
Intervention  ICMJE
  • Drug: glyceryl trierucate
    Glyceryl trierucate is an oil to reduce very long chain fatty acids
    Other Names:
    • Lorenzo's oil
    • GTE
  • Drug: glyceryl trioleate
    Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.
    Other Names:
    • Lorenzo's Oil
    • GTO
Study Arms  ICMJE GTO/GTE treatment
Treatment with GTO/GTE orally; 30-60 ml daily for study period
Interventions:
  • Drug: glyceryl trierucate
  • Drug: glyceryl trioleate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Suspended
Estimated Enrollment  ICMJE
 (submitted: June 23, 2005)
30
Original Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2014
Estimated Primary Completion Date November 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE
  • Biochemically proven asymptomatic X-linked adrenoleukodystrophy
  • Platelet count in normal range
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 18 Months to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00004418
Other Study ID Numbers  ICMJE 199/13312
Nutricia-Loma Linda ( Other Grant/Funding Number: Nutricia-Loma Linda )
KKI-FDR000685 ( Other Identifier: FDA )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Study Sponsor  ICMJE Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gerald V Raymond, M.D. Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
PRS Account Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Verification Date September 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP