A Phase 2 interventional study of glyceryl trierucate/glyceryl trioleate in Adrenoleukodystrophy, sponsored by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.. Terminated at 2 sites in United States. Open to male participants aged 18 Months to 8 Years. Per ClinicalTrials.gov, last updated 2020-10-23.
Sponsored by Hugo W. Moser Research Institute at Kennedy Krieger, Inc. · Phase 2, Interventional, and Treatment
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 126 is above the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Hugo W. Moser Research Institute at Kennedy Krieger, Inc. is the lead sponsor of 76 studies on the registry; 19 are open to participants now.
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Exclusion criteria.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Also known as: Lorenzo's oil
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.
Time frame: Baseline, an average of 10 years, up to age 13
Number of Participants With T2 MRI Abnormality
We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).
Time frame: 10 years
An open trial of Lorenzo's oil spanning decades (1998-2013). Centered at the Kennedy Krieger.
| Milestone | GTO/GTE Treatment |
|---|---|
| Started | 126 |
| Completed | 126 |
| Not completed | 0 |
we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.
| mcg/mL | GTO/GTE Treatment |
|---|---|
| Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels | 0.41 (0.05 to 0.41) |
We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).
| Participants | GTO/GTE Treatment |
|---|---|
| Number of Participants With T2 MRI Abnormality | 0 |
Collected over 10 years (throughout the study). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GTO/GTE Treatment | 1/126 (0.8%) | 3/126 (2.4%) | 1/126 (0.8%) |
| Event | GTO/GTE Treatment |
|---|---|
| adrenal crisisEndocrine disorders | 2/126 |
| DeathEndocrine disorders | 1/126 |
| Event | GTO/GTE Treatment |
|---|---|
| ThrombocytopeniaBlood and lymphatic system disorders | 1/126 |
| Elevation in liver function testsHepatobiliary disorders | 0/126 |
| Age, Categorical(Participants) | GTO/GTE Treatment |
|---|---|
| <=18 years | 126 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | GTO/GTE Treatment |
|---|---|
| Mean | 3.25 (1.5 to 8) |
| Sex: Female, Male(Participants) | GTO/GTE Treatment |
|---|---|
| Female | 0 |
| Male | 126 |
| Region of Enrollment(participants) | GTO/GTE Treatment |
|---|---|
| United States | 126 |
Plan to share: No
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Hugo W. Moser Research Institute at Kennedy Krieger, Inc.