Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

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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

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 Number of Participants With T2 MRI Abnormality

Condition

Adrenoleukodystrophy

Intervention

glyceryl trierucate/glyceryl trioleate

Study Arms / Comparison Groups

 Glyceryl trierucate/glyceryl trioleate
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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

126

Start Date

April 1998

Completion Date

December 2014

Primary Completion Date

December 2014

Eligibility Criteria

        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
      

Gender

Male

Ages

18 Months - 8 Years

Accepts Healthy Volunteers

No

Contacts

Gerald V Raymond, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00004418

Organization ID

199/13312

Secondary IDs

Nutricia-Loma Linda

Responsible Party

Sponsor

Study Sponsor

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


Study Sponsor

Gerald V Raymond, M.D., Principal Investigator, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Verification Date

October 2020