Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
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|ClinicalTrials.gov Identifier: NCT00004418|
Recruitment Status : Terminated (No funding, move to expanded access)
First Posted : October 19, 1999
Results First Posted : October 23, 2020
Last Update Posted : October 23, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|Adrenoleukodystrophy||Drug: glyceryl trierucate/glyceryl trioleate||Phase 2|
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 they reach the age of 13 years or death.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||126 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy|
|Study Start Date :||April 1998|
|Actual Primary Completion Date :||December 2014|
|Actual Study Completion Date :||December 2014|
Experimental: Glyceryl trierucate/glyceryl trioleate
Treatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial
Drug: glyceryl trierucate/glyceryl trioleate
Administration of glyceryl trierucate/glyceryl trioleate
Other Name: Lorenzo's oil
- Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels [ Time Frame: Baseline, an average of 10 years, up to age 13 ]we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.
- Number of Participants With T2 MRI Abnormality [ Time Frame: 10 years ]We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004418
|United States, Maryland|
|Kennedy Krieger Institute|
|Baltimore, Maryland, United States, 21205|
|Johns Hopkins Hospital|
|Baltimore, Maryland, United States, 21287-6681|
|Principal Investigator:||Gerald V Raymond, M.D.||Hugo W. Moser Research Institute at Kennedy Krieger, Inc.|