Trial Outcomes & Findings for Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy (NCT NCT00004418)

Results Overview

we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

126 participants

Primary outcome timeframe

Baseline, an average of 10 years, up to age 13

Results posted on

2020-10-23

An open trial of Lorenzo's oil spanning decades (1998-2013). Centered at the Kennedy Krieger.

Participant milestones
Measure	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.
Overall Study STARTED	126
Overall Study COMPLETED	126
Overall Study NOT COMPLETED	0

Withdrawal data not reported

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Baseline characteristics by cohort
Measure	GTO/GTE Treatment n=126 Participants 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.
Age, Categorical <=18 years	126 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	3.25 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	126 Participants n=5 Participants
Region of Enrollment United States	126 participants n=5 Participants

Timeframe: 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.

Outcome measures
Measure	GTO/GTE Treatment n=126 Participants 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.
Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels	0.41 mcg/mL Interval 0.05 to 0.41

Timeframe: 10 years

We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).

Outcome measures
Measure	GTO/GTE Treatment n=126 Participants 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.
Number of Participants With T2 MRI Abnormality	0 Participants

Serious events: 3 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events
Measure	GTO/GTE Treatment n=126 participants at risk 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.
Endocrine disorders Death	0.79% 1/126 • Number of events 1 • 10 years (throughout the study)
Endocrine disorders adrenal crisis	1.6% 2/126 • Number of events 2 • 10 years (throughout the study)

Other adverse events
Measure	GTO/GTE Treatment n=126 participants at risk 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.
Blood and lymphatic system disorders Thrombocytopenia	0.79% 1/126 • Number of events 1 • 10 years (throughout the study)
Hepatobiliary disorders Elevation in liver function tests	0.00% 0/126 • 10 years (throughout the study)

Kennedy Krieger Institute

Phone: 443-923-2750