NCT00004418

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
126

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Apr 1998

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 1998

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
15.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
5.9 years until next milestone

Results Posted

Study results publicly available

October 23, 2020

Completed
Last Updated

October 23, 2020

Status Verified

October 1, 2020

Enrollment Period

16.7 years

First QC Date

October 18, 1999

Results QC Date

April 13, 2016

Last Update Submit

October 22, 2020

Conditions

Adrenoleukodystrophy

Keywords

adrenoleukodystrophyinborn errors of metabolismrare diseasesphingolipidoses

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (1)

  • Number of Participants With T2 MRI Abnormality

    10 years

Study Arms (1)

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

Drug: glyceryl trierucate/glyceryl trioleate

Interventions

glyceryl trierucate/glyceryl trioleateDRUG

Administration of glyceryl trierucate/glyceryl trioleate

Also known as: Lorenzo's oil
Glyceryl trierucate/glyceryl trioleate

Eligibility Criteria

Age18 Months - 8 Years
Sexmale(Gender-based eligibility)
Gender Eligibility Detailsmales only
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Boys between the age of 18 months and 8 years of age
  • Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.
  • Platelet count in normal range

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287-6681, United States

Location

MeSH Terms

Conditions

AdrenoleukodystrophyMetabolism, Inborn ErrorsRare DiseasesSphingolipidoses

Interventions

trierucateTrioleinLorenzo's oil

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHereditary Central Nervous System Demyelinating DiseasesLeukoencephalopathiesDemyelinating DiseasesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemPeroxisomal DisordersMetabolic DiseasesNutritional and Metabolic DiseasesAdrenal InsufficiencyAdrenal Gland DiseasesEndocrine System DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLysosomal Storage Diseases, Nervous SystemLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesLipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Fats, UnsaturatedFatsLipidsTriglyceridesGlycerides

Results Point of Contact

Title
Gerald Raymond
Organization
Kennedy Krieger Institute
raymond@kennedykrieger.org
443-923-2750

Study Officials

  • Gerald V Raymond, M.D.

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

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

April 1, 1998

Primary Completion

December 1, 2014

Study Completion

December 1, 2014

Last Updated

October 23, 2020

Results First Posted

October 23, 2020

Record last verified: 2020-10

Data Sharing

IPD Sharing
Will not share

Locations

US(2)