Expired Study
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Baltimore, Maryland 21287


Purpose:

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.


Study summary:

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.


Criteria:

- Biochemically proven asymptomatic X-linked adrenoleukodystrophy - Platelet count in normal range


Study is Available At:


Original ID:

199/13312


NCT ID:

NCT00004418


Secondary ID:

Nutricia-Loma Linda


Study Acronym:


Brief Title:

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy


Official Title:

Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy


Overall Status:

Suspended


Study Phase:

Phase 2/Phase 3


Genders:

Male


Minimum Age:

18 Months


Maximum Age:

6 Years


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Oversight Authority:

United States: Food and Drug Administration


Reasons Why Stopped:

No funding and moved to expanded access


Study Type:

Interventional


Study Design:

Endpoint Classification: Safety/Efficacy Study, In


Number of Arms:

1


Number of Groups:

0


Total Enrollment:

30


Enrollment Type:

Anticipated


Overall Contact Information

Official Name:Gerald V Raymond, M.D.
Principal Investigator
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Study Dates

Start Date:April 1998
Completion Date:November 2014
Completion Type:Anticipated
Primary Completion Date:November 2014
Primary Completion Type:Anticipated
Verification Date:September 2014
Last Changed Date:September 17, 2014
First Received Date:October 18, 1999

Study Outcomes

Outcome Type:Primary Outcome
Measure:neurological disability
Time Frame:6 months post intervention, then every 3 months until age 13 years
Safety Issues:False
Outcome Type:Secondary Outcome
Measure:magnetic resonance imaging of the head
Time Frame:yearly
Safety Issues:False

Study Interventions

Intervention Type:Drug
Name:glyceryl trierucate
Description:Glyceryl trierucate is an oil to reduce very long chain fatty acids
Arm Name:GTO/GTE treatment
Other Name:Lorenzo's oil
Intervention Type:Drug
Name:glyceryl trioleate
Description:Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.
Arm Name:GTO/GTE treatment
Other Name:Lorenzo's Oil

Study Arms

Study Arm Type:Other
Arm Name:GTO/GTE treatment
Description:Treatment with GTO/GTE orally; 30-60 ml daily for study period

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: January 21, 2020

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