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| ID | Type | Description | Link |
|---|---|---|---|
| Nutricia-Loma Linda | Other Grant/Funding Number | Nutricia-Loma Linda | |
| KKI-FDR000685 | Other Identifier | FDA |
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No funding, move to expanded access
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Glyceryl trierucate/glyceryl trioleate | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| glyceryl trierucate/glyceryl trioleate | Drug | Administration of glyceryl trierucate/glyceryl trioleate |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion criteria.
Exclusion criteria.
males only
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| Name | Affiliation | Role |
|---|---|---|
| Gerald V Raymond, M.D. | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States | ||
| Johns Hopkins Hospital |
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An open trial of Lorenzo's oil spanning decades (1998-2013). Centered at the Kennedy Krieger.
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| ID | Title | Description |
|---|---|---|
| FG000 | GTO/GTE Treatment | Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats. GTO/GTE orally; 30-60 ml daily for study period glyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids glyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | GTO/GTE Treatment | Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats. GTO/GTE orally; 30-60 ml daily for study period glyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids glyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Mean | Full Range | mcg/mL | Baseline, an average of 10 years, up to age 13 |
|
10 years (throughout the study)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GTO/GTE Treatment | Intervention: A mixture of glycerol trierucate (GTE) in glycerol trioleate (GTO) in combination with a diet low in saturated fats. GTO/GTE orally; 30-60 ml daily for study period glyceryl trierucate: Glyceryl trierucate is an oil to reduce very long chain fatty acids glyceryl trioleate: Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Death | Endocrine disorders | Non-systematic Assessment | Subject had profound adrenal crisis and died |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | Non-systematic Assessment | Low platelet count defined as <80, 000 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Gerald Raymond | Kennedy Krieger Institute | 443-923-2750 | raymond@kennedykrieger.org |
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| ID | Term |
|---|---|
| D000326 | Adrenoleukodystrophy |
| D008661 | Metabolism, Inborn Errors |
| D035583 | Rare Diseases |
| D013106 | Sphingolipidoses |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C030774 | trierucate |
| D014304 | Triolein |
| C079420 | Lorenzo's oil |
| ID | Term |
|---|---|
| D005224 | Fats, Unsaturated |
| D005223 | Fats |
| D008055 | Lipids |
| D014280 | Triglycerides |
| D005989 |
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| Baltimore |
| Maryland |
| 21287-6681 |
| United States |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Male children only were eligible for this study. | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| 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). | Posted | Count of Participants | Participants | 10 years |
|
|
|
| 1 |
| 126 |
| 3 |
| 126 |
| 1 |
| 126 |
|
| adrenal crisis | Endocrine disorders | Non-systematic Assessment | Two events of adrenal crisis |
|
|
| Elevation in liver function tests | Hepatobiliary disorders | Non-systematic Assessment |
|
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| D009422 | Nervous System Diseases |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D018901 | Peroxisomal Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000309 | Adrenal Insufficiency |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D052439 | Lipid Metabolism Disorders |
| Glycerides |