Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Adrenoleukodystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00004418
• Other study ID #: 199/13312
• Secondary ID: Nutricia-Loma Li
• Status: Terminated
• Phase: Phase 2
• Start date: April 1998
• Est. completion date: December 2014

Study information

• Verified date: October 2020
• Source: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 126
• Est. completion date: December 2014
• Est. primary completion date: December 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Months to 8 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy

Intervention

Drug:
• glyceryl trierucate/glyceryl trioleate
Administration of glyceryl trierucate/glyceryl trioleate

Locations

Country	Name	City	State
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	Kennedy Krieger Institute	Baltimore	Maryland

Sponsors (1)

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

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels	we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.	Baseline, an average of 10 years, up to age 13
Secondary	Number of Participants With T2 MRI Abnormality	We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).	10 years