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Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

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.
 
ClinicalTrials.gov Identifier: NCT00004450
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
Sponsor:
Collaborator:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Information provided by:
FDA Office of Orphan Products Development

Tracking Information
First Submitted Date  ICMJE October 18, 1999
First Posted Date  ICMJE October 19, 1999
Last Update Posted Date March 25, 2015
Study Start Date  ICMJE August 1998
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Official Title  ICMJE Not Provided
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.

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Masking: Double
Primary Purpose: Treatment
Condition  ICMJE Adrenoleukodystrophy
Intervention  ICMJE
  • Drug: glyceryl trierucate
  • Drug: glyceryl trioleate
  • Drug: interferon beta
  • Drug: thalidomide
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE
 (submitted: June¬†23,¬†2005)
60
Original Enrollment  ICMJE Same as current
Study Completion Date  ICMJE November 2000
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00004450
Other Study ID Numbers  ICMJE 199/13532
KKI-94-06-16-01
KKI-FDR001052
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE FDA Office of Orphan Products Development
Collaborators  ICMJE Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Investigators  ICMJE
Study Chair: Hugo Wolfgang Moser Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
PRS Account FDA Office of Orphan Products Development
Verification Date January 2001

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP