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Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004450
First Posted: October 19, 1999
Last Update Posted: March 25, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Information provided by:
FDA Office of Orphan Products Development
  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
Masking: Double
Primary Purpose: Treatment

Resource links provided by NLM:


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

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Sexes 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

ClinicalTrials.gov Identifier: NCT00004450     History of Changes
Other Study ID Numbers: 199/13532
KKI-94-06-16-01
KKI-FDR001052
First Submitted: October 18, 1999
First Posted: October 19, 1999
Last Update Posted: March 25, 2015
Last Verified: January 2001

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

Additional relevant MeSH terms:
Adrenoleukodystrophy
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
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases
Interferons
Thalidomide
Interferon-beta
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors