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VK0214 · 1 trial · 1 indication
Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN
| Arm | Type | Description |
|---|---|---|
| VK0214 Active 20mg | EXPERIMENTAL | 20mg QD |
| VK0214 Active 40mg | EXPERIMENTAL | 40mg QD |
| Placebo | PLACEBO_COMPARATOR | Placebo QD |
| Name | Type | Description |
|---|---|---|
| VK0214 | DRUG | API in capsule |
| Placebo | OTHER | API in capsule |
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...
VK0214 is an investigational small molecule being developed for adrenomyeloneuropathy, the AMN form of X-linked adrenoleukodystrophy (X-ALD), a rare inherited neurological disorder. It is in Phase 1 clinical development and is not approved for any indication.
VK0214 targets thyroid hormone receptor beta, a nuclear receptor. By acting on this receptor, the small molecule is designed to modulate thyroid hormone signaling pathways relevant to the metabolic and neurological features of adrenomyeloneuropathy in X-linked adrenoleukodystrophy.
VK0214 is being developed by Viking Therapeutics, Inc., which trades on the Nasdaq under the ticker VKTX. The company is advancing the compound for adrenomyeloneuropathy, the AMN form of X-linked adrenoleukodystrophy.
VK0214 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The completed Phase 1 study evaluated its pharmacodynamics in adult male subjects with adrenomyeloneuropathy.
VK0214 has been studied in one Phase 1 trial, NCT04973657, titled A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN. The completed trial enrolled 24 participants across the United States, France, Germany, Italy, and the United Kingdom.
The Phase 1 trial NCT04973657 was a randomized, double-blind, placebo-controlled study of VK0214 in 24 adult male participants with adrenomyeloneuropathy. It enrolled participants in the United States, France, Germany, Italy, and the United Kingdom and has been completed.