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Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Primary Purpose

Adrenoleukodystrophy

Status

Terminated

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

glyceryl trierucate/glyceryl trioleate

About this trial

This is an interventional treatment trial for Adrenoleukodystrophy focused on measuring adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses

Eligibility Criteria

18 Months - 8 Years (Child)MaleDoes not accept healthy volunteers

Inclusion criteria. Boys between the age of 18 months and 8 years of age Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis. Platelet count in normal range Exclusion criteria. Abnormal MRI consistent with childhood cerebral disease Boys who have undergone bone marrow transplantation Other medical condition which in the opinion of the investigator prevents evaluation or treatment

Sites / Locations

Kennedy Krieger Institute
Johns Hopkins Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Glyceryl trierucate/glyceryl trioleate

Arm 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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Number of Participants With T2 MRI Abnormality

We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).

Full Information

NCT ID

NCT00004418

First Posted

October 18, 1999

Last Updated

October 22, 2020

Sponsor

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

1. Study Identification

Unique Protocol Identification Number

NCT00004418

Brief Title

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2020

Overall Recruitment Status

Terminated

Why Stopped

No funding, move to expanded access

Study Start Date

April 1998 (undefined)

Primary Completion Date

December 2014 (Actual)

Study Completion Date

December 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

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

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Adrenoleukodystrophy

Keywords

adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

126 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Glyceryl trierucate/glyceryl trioleate

Arm Type

Experimental

Arm 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

Intervention Type

Drug

Intervention Name(s)

glyceryl trierucate/glyceryl trioleate

Other Intervention Name(s)

Lorenzo's oil

Intervention Description

Administration of glyceryl trierucate/glyceryl trioleate

Primary Outcome Measure Information:

Title

Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels

Description

we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.

Time Frame

Baseline, an average of 10 years, up to age 13

Secondary Outcome Measure Information:

Title

Number of Participants With T2 MRI Abnormality

Description

We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).

Time Frame

10 years

10. Eligibility

Sex

Male

Gender Based

Yes

Gender Eligibility Description

males only

Minimum Age & Unit of Time

18 Months

Maximum Age & Unit of Time

8 Years

Accepts Healthy Volunteers

Eligibility Criteria

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Gerald V Raymond, M.D.

Organizational Affiliation

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

Official's Role

Principal Investigator

Facility Information:

Facility Name

Kennedy Krieger Institute

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Johns Hopkins Hospital

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21287-6681

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

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