Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

October 22, 2020 updated by: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

126

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
      • Baltimore, Maryland, United States, 21287-6681
        • Johns Hopkins Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 8 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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
Drug: glyceryl trierucate/glyceryl trioleate
Administration of glyceryl trierucate/glyceryl trioleate
Other Names:
  • Lorenzo's oil

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels
Time Frame: 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.
Baseline, an average of 10 years, up to age 13

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With T2 MRI Abnormality
Time Frame: 10 years
We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 1998

Primary Completion (Actual)

December 1, 2014

Study Completion (Actual)

December 1, 2014

Study Registration Dates

First Submitted

October 18, 1999

First Submitted That Met QC Criteria

October 18, 1999

First Posted (Estimate)

October 19, 1999

Study Record Updates

Last Update Posted (Actual)

October 23, 2020

Last Update Submitted That Met QC Criteria

October 22, 2020

Last Verified

October 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 199/13312
  • Nutricia-Loma Linda (Other Grant/Funding Number: Nutricia-Loma Linda)
  • KKI-FDR000685 (Other Identifier: FDA)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Adrenoleukodystrophy

Clinical Trials on glyceryl trierucate/glyceryl trioleate

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ethiopia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe