A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN (NCT04973657) | Clinical Trial Compass
CompletedPhase 1
A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN
United States, France24 participantsStarted 2021-06-22
Plain-language summary
Subjects will be administered multiple doses of VK0214 in a cohort dose escalation process. A total of up to 36 subjects will be enrolled in the study, into one of three dose cohorts. For each cohort, up to 12 subjects will be randomized to receive VK0214 or placebo in a 3:1 ratio so that there will be a total of up to 9 subjects for each of the active doses and up to 3 subjects dosed with placebo in each cohort.
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* Provide a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study and is willing and able to participate.
* Have diagnosis of X-linked adrenoleukodystrophy (X-ALD) by genetic testing and elevated VLCFAs.
* Present clinical features of AMN, or adrenal insufficiency.
* Subjects must be 18 years of age and older.
Exclusion Criteria:
* Have the cerebral form of X-ALD, based on medical records, or based on MRI performed within the last 12 months or MRI at screening
* Have been treated with any drug targeting TRβ or with any drug being tested as therapy 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 or oncological that in the opinion of the investigator would pose a significant risk for the subject
* Subject has untreated primary adrenal insufficiency based on morning plasma cortisol and ACTH at screening
* Lorenzo's Oil. Currently taking Lorenzo's Oil or erucic acid containing product, or other lipid lowering agent known to effect VLCFA levels
What they're measuring
1
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs)