Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Phase 2

Terminated

• Conditions: Adrenoleukodystrophy
• Interventions: Drug: glyceryl trierucate/glyceryl trioleate

• Registration Number: NCT00004418
• Lead Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Brief 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.

Study Timeline

• Study Start: 1998-04
• Study First Submitted: 1999-10-18
• Study First Submitted QC: 1999-10-18
• Study First Posted: 1999-10-19
• Primary Completion: 2014-12
• Study Completion: 2014-12
• Results First Submitted: 2016-04-13
• Status Verified: 2020-10
• Results First Submitted QC: 2020-10-22
• Last Update Submitted: 2020-10-22
• Results First Posted: 2020-10-23
• Last Update Posted: 2020-10-23

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 126

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Glyceryl trierucate/glyceryl trioleate	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

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels	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.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With T2 MRI Abnormality	10 years	We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).

Trial Locations

Locations (2)

Johns Hopkins Hospital; 🇺🇸 Baltimore, Maryland, United States

Kennedy Krieger Institute; 🇺🇸 Baltimore, Maryland, United States