Participation in this study is limited to patients with x-linked adrenoleukodystrophy who have evidence of a brain inflammatory response on the basis of gradolinium magnetic resonance imaging studies, and whose neurologic involvement is too advanced for consideration of bone marrow transplant. The double blinded placebo-controlled study wims to determine whether progression of the inflammatory cascade can be interrupted by beta interferon or by thalidomide. Twenty one patients have been enrolled in this demanding protocol volumetric magnetic reasonance image analysis. Except for skin rashes in two patients no significant side effects have been observed. While the majority of the patients with this extremely grave form of ALD have continued to lose ground neurologically, the data suggest that some of the patients have sstabilized. The code has not been broken. The study is being monitored by an unblinded data management and monitoring committee. The committee has advised us that no statistically significant differences hae emerged between the three groups with the limited data available at this time and has advised us to continue the study. We anticipate to enroll an additional 20 patients during the next year.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
3M01RR000052-38S1
Application #
6297480
Study Section
Project Start
1998-12-01
Project End
1999-11-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
38
Fiscal Year
1999
Total Cost
Indirect Cost
Name
Johns Hopkins University
Department
Type
DUNS #
045911138
City
Baltimore
State
MD
Country
United States
Zip Code
21218
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