Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

Adrenoleukodystrophy Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004450
• Other study ID #: 199/13532
• Secondary ID: KKI-94-06-16-01K
• Status: Completed
• Phase: N/A
• First received: October 18, 1999
• Last updated: March 24, 2015
• Start date: August 1998
• Est. completion date: November 2000

Study information

• Verified date: January 2001
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: November 2000
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Adrenoleukodystrophy

Intervention

Drug:
• glyceryl trierucate

• glyceryl trioleate

• interferon beta

• thalidomide

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
FDA Office of Orphan Products Development	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.