Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 7 April 2015
• Main ID: NCT00004450
• Date of registration: 18/10/1999
• Prospective Registration: No
• Primary sponsor: FDA Office of Orphan Products Development
• Public title: Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
• Date of first enrolment: August 1998
• Target sample size: 60
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00004450
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Masking: Double-Blind, Primary Purpose: Treatment
• Phase: N/A

Contacts

• Name: Hugo Wolfgang Moser
• Affiliation: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Key inclusion & exclusion 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



Age minimum: 4 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Adrenoleukodystrophy

Intervention(s)

Drug: interferon beta

Drug: glyceryl trierucate

Drug: glyceryl trioleate

Drug: thalidomide

Secondary ID(s)

199/13532

KKI-FDR001052

KKI-94-06-16-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.