Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

NCT00004450 | Completed

• 3 other identifiers: 199/13532KKI-94-06-16-01KKI-FDR001052
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief Summary

OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil). II. Evaluate the progress of the disease and possible side effects of the medication in these patients.

Conditions

Adrenoleukodystrophy

Keywords

adrenoleukodystrophy; inborn errors of metabolism; rare disease; sphingolipidoses

Interventions

glyceryl trierucate DRUG

glyceryl trioleate DRUG

interferon beta DRUG

thalidomide DRUG

Eligibility Criteria

• Age: 4 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Adrenoleukodystrophy (ALD) diagnosis based on history and examination, MRI, and biochemical assay Clinical evidence of rapidly progressive phase of cerebral ALD must include 2 or more of the following during the preceding year: Significant and progressive impairment of school performance Significant loss of cognitive function leading to an IQ of 75 or less Progressive impairment of the ability to understand spoken words Progressive impairment of vision Progressive deterioration of handwriting Progressive difficulty in walking Progressive impairment in speech articulation, and vocabulary Progressive weakness of one or more limbs Must have MRI abnormalities characteristic of cerebral ALD, especially evidence of the breakdown of the blood-brain barrier using gadolinium contrast medium and magnetization transfer technique Evidence of brain white matter inflammatory response Must not meet criteria for bone marrow transplantation Not in an apparent vegetative state --Prior/Concurrent Therapy-- Concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy required --Patient Characteristics-- Effective contraception required of all patients

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• FDA Office of Orphan Products Development: lead
• Hugo W. Moser Research Institute at Kennedy Krieger, Inc.: collaborator

MeSH Terms

Conditions

Adrenoleukodystrophy; Metabolism, Inborn Errors; Rare Diseases; Sphingolipidoses

Interventions

trierucate; Triolein; Interferon-beta; Thalidomide

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Fats, Unsaturated; Fats; Lipids; Triglycerides; Glycerides; Interferon Type I; Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Hugo Wolfgang Moser

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

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Sponsor Type: FED

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: August 1, 1998
• Study Completion: November 1, 2000
• Last Updated: March 25, 2015

Record last verified: 2001-01