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Clinical Trial NCT04973657 for Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) is completed. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here.
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A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN Phase 1 24 Randomized Dose Escalation

Completed
Clinical Trial NCT04973657 was designed to study Other for Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD). This was a Phase 1 interventional study that is now completed. The study started on June 22, 2021, with plans to enroll 24 participants. Led by Viking Therapeutics, Inc., the expected completion date was November 8, 2024. The latest data from ClinicalTrials.gov was last updated on February 17, 2025.
Brief 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.
Detailed Description
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 ...

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Official Title

Phase 1b, Multi-center, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability, PD and PK of VK0214, in Subjects With the Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

Conditions
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
Other Study IDs
  • VK0214-102
NCT ID Number
NCT04973657
Start Date (Actual)
2021-06-22
Last Update Posted
2025-02-17
Completion Date (Estimated)
2024-11-08
Enrollment (Estimated)
24
Study Type
Interventional
PHASE
Phase 1
Status
Completed
Keywords
AMN, XALD
Primary Purpose
Other
Design Allocation
Randomized
Interventional Model
Sequential
Masking
Quadruple
Arms / Interventions
Participant Group/ArmIntervention/Treatment
ExperimentalVK0214 Active 20mg
20mg QD
VK0214
API in capsule
ExperimentalVK0214 Active 40mg
40mg QD
VK0214
API in capsule
Placebo ComparatorPlacebo
Placebo QD
Placebo
API in capsule
Primary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
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
28 days
Secondary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Evaluate the Pharmacokinetics of VK0214
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)
28 days
Eligibility Criteria

Eligible Ages
Adult, Older Adult
Minimum Age
18 Years
Eligible Sexes
All
  • 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.

  • 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
Viking Therapeutics, Inc. logoViking Therapeutics, Inc.
No contact data.
9 Study Locations in 5 Countries
Viking Clinical Site 212, London, WC1N3BG, United Kingdom
Viking Clinical Site 209, Leipzig, 04103, Germany
Viking Clinical Site 214, Bordeaux, 33076, France
Viking Clinical Site 215, Montpellier, 34295, France

California

Viking Clinical Site 203, Palo Alto, California, 94303, United States

Florida

Viking Clinical Site 207, Gainesville, Florida, 32610, United States

Maryland

Viking Clinical Site 201, Baltimore, Maryland, 21205, United States

Washington

Viking Clinical Site 205, Seattle, Washington, 98195, United States
Viking Clinical Site 210, Milan, 20133, Italy