NCT00004418

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 126 (actual)
• Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc. · Academic / Other
• Sex: Male
• Age: 18 Months – 8 Years
• Healthy volunteers: Not accepted

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 they reach the age of 13 years or death.

Conditions

• Adrenoleukodystrophy

Interventions

Type	Name	Description
DRUG	glyceryl trierucate/glyceryl trioleate	Administration of glyceryl trierucate/glyceryl trioleate

Timeline

• Start date: 1998-04-01
• Primary completion: 2014-12-01
• Completion: 2014-12-01
• First posted: 1999-10-19
• Last updated: 2020-10-23
• Results posted: 2020-10-23

Locations

2 sites across 1 country: United States