Phase
Condition
Bone Marrow Disorder
Treatment
Placebo
VK0214
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Provide a personally signed and dated informed consent document indicating that thesubject has been informed of all pertinent aspects of the study and is willing andable to participate.
Have diagnosis of X-linked adrenoleukodystrophy (X-ALD) by genetic testing andelevated VLCFAs.
Present clinical features of AMN, or adrenal insufficiency.
Subjects must be 18 years of age and older.
Exclusion
Exclusion Criteria:
Have the cerebral form of X-ALD, based on medical records, or based on MRI performedwithin the last 12 months or MRI at screening
Have been treated with any drug targeting TRβ or with any drug being tested astherapy for X-ALD AMN form within the past 30 days before screening
History or presence of clinically significant acute or unstable hepatic, renal,gastrointestinal, pulmonary, immunological, endocrine, diabetes, hematological oroncological that in the opinion of the investigator would pose a significant riskfor the subject
Subject has untreated primary adrenal insufficiency based on morning plasma cortisoland ACTH at screening
Lorenzo's Oil. Currently taking Lorenzo's Oil or erucic acid containing product, orother lipid lowering agent known to effect VLCFA levels
Study Design
Study Description
Connect with a study center
Viking Clinical Site 214
Bordeaux, 33076
FranceSite Not Available
Viking Clinical Site 215
Montpellier, 34295
FranceSite Not Available
Viking Clinical Site 209
Leipzig, 04103
GermanySite Not Available
Viking Clinical Site 210
Milano, 20133
ItalySite Not Available
Viking Clinical Site 212
London, WC1N3BG
United KingdomSite Not Available
Viking Clinical Site 203
Palo Alto, California 94303
United StatesSite Not Available
Viking Clinical Site 207
Gainesville, Florida 32610
United StatesSite Not Available
Viking Clinical Site 201
Baltimore, Maryland 21205
United StatesSite Not Available
Viking Clinical Site 206
New York, New York 10021
United StatesSite Not Available
Viking Clinical Site 205
Seattle, Washington 98195
United StatesSite Not Available
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