A Phase 1 interventional study of VK0214 and Placebo in Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD), sponsored by Viking Therapeutics, Inc.. Completed at 9 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-02-17.
Sponsored by Viking Therapeutics, Inc. · Phase 1, Interventional, and Other
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.
The first part of VK0214-102 will include the first 2 dose cohorts and placebo, which will be dosed in parallel (up to N=24). The first 2 doses used in these cohorts will be 20 mg QD and 40 mg QD for 28 days. A Dose Level Review Team (DLRT) meeting will be held at the end of the first 2 parallel doses.
The DLRT will review safety, tolerability, and preliminary efficacy data from cohort 1 and 2. If the data analyzed show that the first 2 doses are safe and well tolerated the DLRT may make a recommendation for proceeding to commence dosing in the third dose cohort.
Based upon outcomes in Cohorts 1 and 2, the sponsor may decide to include a third dose cohort. Before screening may be initiated in Cohort 3, data to support dosing in this cohort will be submitted to the FDA, together with the amended protocol and the dosing recommendation of the DLRT.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 24 is below the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Viking Therapeutics, Inc. is the lead sponsor of 13 studies on the registry; 4 are open to participants now.
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Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
20mg QD
Drug: VK0214
40mg QD
Drug: VK0214
Placebo QD
Other: Placebo
API in capsule
API in capsule
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs)
Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN
Time frame: 28 days
Evaluate the Pharmacokinetics of VK0214
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)
Time frame: 28 days
Evaluate plasma VLCFAs changes
Evaluate the efficacy of VK0214 on lowering plasma levels of VLCFAs in subjects with AMN
Time frame: 28 days
Plan to share: No
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This study is completed, as verified in Feb 2025. You cannot join it, but the record below documents what was studied.
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