Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

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

Status and phase: Terminated; Phase 2

Conditions: Adrenoleukodystrophy

Treatments: Drug: glyceryl trierucate/glyceryl trioleate

Study type: Interventional

Funder types: Other

Identifiers

NCT00004418

KKI-FDR000685 (Other Identifier)

Nutricia-Loma Linda (Other Grant/Funding Number)

199/13312

Details and patient eligibility

About

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.

Full 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 they reach the age of 13 years or death.

Enrollment: 126 patients

Sex: Male

Ages: 18 months to 8 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion criteria.

1. Boys between the age of 18 months and 8 years of age
2. Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.
3. Platelet count in normal range

Exclusion criteria.

1. Abnormal MRI consistent with childhood cerebral disease
2. Boys who have undergone bone marrow transplantation
3. Other medical condition which in the opinion of the investigator prevents evaluation or treatment

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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126 participants in 1 patient group

Glyceryl trierucate/glyceryl trioleate

Experimental group

Description:

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

Treatment:

Drug: glyceryl trierucate/glyceryl trioleate