Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

• Status: Terminated
• Trial end date: 2014-12-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

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