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Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

This study has been completed.

Sponsors and Collaborators: FDA Office of Orphan Products Development
Kennedy-Krieger Research Institute
Information provided by: FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier: NCT00004450
  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

Genetics Home Reference related topics:   beta-ketothiolase deficiency    familial encephalopathy with neuroserpin inclusion bodies    L1 syndrome    leukoencephalopathy with vanishing white matter    X-linked adrenoleukodystrophy   

MedlinePlus related topics:   Addison's Disease   

ChemIDplus related topics:   Thalidomide    Interferon alfa-2b    Interferons    Interferon beta    Interferon-beta    Triolein   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized, Double-Blind, Placebo Control, Efficacy Study

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
Kennedy-Krieger Research Institute

Investigators
Study Chair:     Hugo Wolfgang Moser     Kennedy-Krieger Research Institute    
  More Information


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

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

Study placed in the following topic categories:
Sphingolipidoses
Addison's disease
Thalidomide
Adrenal Gland Diseases
Demyelinating diseases
Hypoadrenalism
Brain Diseases
Metabolism, Inborn Errors
Heredodegenerative Disorders, Nervous System
Peroxisomal Disorders
Adrenoleukodystrophy
Addison Disease
Genetic Diseases, X-Linked
Brain Diseases, Metabolic, Inborn
Neurobehavioral Manifestations
X-linked adrenoleukodystrophy
Interferon-alpha
Adrenal Insufficiency
Metabolic Diseases
Autoimmune Diseases
Demyelinating Diseases
Interferons
Interferon-beta
Rare Diseases
Central Nervous System Diseases
Endocrine System Diseases
Sphingolipidosis
Adrenal gland hypofunction
Mental Retardation
Genetic Diseases, Inborn

Additional relevant MeSH terms:
Anti-Infective Agents
Immune System Diseases
Immunologic Factors
Antineoplastic Agents
Growth Substances
Nervous System Diseases
Physiological Effects of Drugs
Immunosuppressive Agents
Hereditary Central Nervous System Demyelinating Diseases
Antiviral Agents
Angiogenesis Inhibitors
Pharmacologic Actions
Anti-Bacterial Agents
Therapeutic Uses
Growth Inhibitors
Angiogenesis Modulating Agents
Mental Retardation, X-Linked
Leprostatic Agents

ClinicalTrials.gov processed this record on October 10, 2008




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