Suspended

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

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What is being tested

glyceryl trierucate/glyceryl trioleate

Drug
Who is being recruted

Adrenal Gland Diseases+31

+ Adrenal Insufficiency

+ Brain Diseases

From 18 Months to 8 Years
+6 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: April 1998
See protocol details

Summary

Principal SponsorHugo W. Moser Research Institute at Kennedy Krieger, Inc.
Last updated: October 23, 2020
Sourced from a government-validated database.Claim as a partner

Study start date: April 1, 1998

Actual date on which the first participant was enrolled.

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.

NCT00004418
Principal SponsorHugo W. Moser Research Institute at Kennedy Krieger, Inc.
Last updated: October 23, 2020
Sourced from a government-validated database.Claim as a partner

Protocol

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

126 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Male

Biological sex of participants that are eligible to enroll.

From 18 Months to 8 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Adrenal Gland DiseasesAdrenal InsufficiencyBrain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesDemyelinating DiseasesEndocrine System DiseasesLipid Metabolism, Inborn ErrorsLipidosesIntellectual DisabilityMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsNutritional and Metabolic DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsLysosomal Storage DiseasesPeroxisomal DisordersNeurobehavioral ManifestationsLysosomal Storage Diseases, Nervous SystemHeredodegenerative Disorders, Nervous SystemHereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornDisease AttributesGenetic Diseases, InbornX-Linked Intellectual DisabilityGenetic Diseases, X-LinkedLipid Metabolism DisordersLeukoencephalopathiesAdrenoleukodystrophyMetabolism, Inborn ErrorsSphingolipidosesRare Diseases

Criteria

3 inclusion criteria required to participate
Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis
Boys between the age of 18 months and 8 years of age
Platelet count in normal range
3 exclusion criteria prevent from participating
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

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
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

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 2 locations

Kennedy Krieger Institute

Baltimore, United StatesOpen Kennedy Krieger Institute in Google Maps

Johns Hopkins Hospital

Baltimore, United States
Suspended2 Study Centers