VK0214 for Adrenoleukodystrophy

Recruiting in Palo Alto (17 mi)

+9 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Viking Therapeutics, Inc.

Trial Summary

What is the purpose of this trial?

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.

Research Team

Eligibility Criteria

This trial is for adult males with X-linked adrenoleukodystrophy (X-ALD) who show symptoms of adrenomyeloneuropathy (AMN) or adrenal insufficiency, confirmed by genetic testing and elevated VLCFAs. Participants must be informed about the study and willing to join. Those using certain lipid-lowering agents, with untreated primary adrenal insufficiency, cerebral X-ALD, or significant acute health issues are excluded.

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

You have been diagnosed with X-linked adrenoleukodystrophy (X-ALD) through genetic testing and have high levels of VLCFAs.

Subjects must be 18 years of age and older

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Exclusion Criteria

You are currently taking Lorenzo's Oil, erucic acid-containing products, or any other medication that can affect certain lipid levels.

You have a specific condition called primary adrenal insufficiency that has not been treated yet.

You have a serious ongoing health condition that could put you at risk during the study, like liver, kidney, digestive, lung, immune, hormone, blood, or cancer problems.

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Treatment Details

Interventions

VK0214 (Steroid)

Trial OverviewThe study tests VK0214's effects on AMN in a dose escalation format across three groups. Each group has up to 12 men taking either VK0214 or placebo at a 3:1 ratio. The goal is to find out how different doses of VK0214 affect the body compared to a placebo.

Participant Groups

3Treatment groups

Experimental Treatment

Placebo Group

Group I: VK0214 Active 40mgExperimental Treatment1 Intervention

40mg QD

Group II: VK0214 Active 20mgExperimental Treatment1 Intervention

20mg QD

Group III: PlaceboPlacebo Group1 Intervention

Placebo QD