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Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

This study has been completed.
Sponsor:
Collaborator:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004450
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: January 2001
History of Changes
  Purpose

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.


Condition Intervention
Adrenoleukodystrophy
 Drug: glyceryl trierucate
Drug: glyceryl trioleate
Drug: interferon beta
Drug: thalidomide

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 60
Study Start Date: August 1998
Estimated Study Completion Date: November 2000
Detailed Description:

PROTOCOL OUTLINE: This is randomized, double blind, placebo controlled study. Patients are randomized to receive beta interferon and thalidomide placebo (arm I), thalidomide and beta interferon placebo (arm II), or placebo for both beta interferon and thalidomide (arm III). Patients receive interferon beta by subcutaneous injection and thalidomide orally. All patients are maintained on glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy.

Patients are followed at 3, 6, and 12 months and then may be followed every 6 months thereafter.

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004450

Sponsors and Collaborators
FDA Office of Orphan Products Development
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Investigators
Study Chair: Hugo Wolfgang Moser Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004450     History of Changes
Other Study ID Numbers: 199/13532, KKI-94-06-16-01, KKI-FDR001052
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
adrenoleukodystrophy
inborn errors of metabolism
rare disease
sphingolipidoses

Additional relevant MeSH terms:
Adrenoleukodystrophy
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Peroxisomal Disorders
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
 Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases
Interferon-beta
Interferons
Thalidomide
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Immunologic Factors

ClinicalTrials.gov processed this record on May 22, 2013