Ignite Creation Date:
2025-12-25 @ 4:42 AM
Ignite Modification Date:
2025-12-26 @ 3:44 AM
Study NCT ID:
NCT00004418
Status:
TERMINATED
Last Update Posted:
2020-10-23
First Post:
1999-10-18
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Sponsor:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Organization:
Study Overview
Official Title:
Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
Status:
TERMINATED
Status Verified Date:
2020-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
No funding, move to expanded access
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.
Detailed Description:
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.
Patients are followed monthly for 6 months, then every 3 months until they reach the age of 13 years or death.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: