A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

February 13, 2025 updated by: Viking Therapeutics, Inc.

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 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.

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Bordeaux, France, 33076
        • Viking Clinical Site 214
      • Montpellier, France, 34295
        • Viking Clinical Site 215
  • Germany
      • Leipzig, Germany, 04103
        • Viking Clinical Site 209
  • Italy
      • Milano, Italy, 20133
        • Viking Clinical Site 210
  • United Kingdom
      • London, United Kingdom, WC1N3BG
        • Viking Clinical Site 212
  • United States
    • California
      • Palo Alto, California, United States, 94303
        • Viking Clinical Site 203
    • Florida
      • Gainesville, Florida, United States, 32610
        • Viking Clinical Site 207
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Viking Clinical Site 201
    • Washington
      • Seattle, Washington, United States, 98195
        • Viking Clinical Site 205

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Placebo QD
Other: Placebo
API in capsule
Experimental: VK0214 Active 20mg
20mg QD
Drug: VK0214
API in capsule
Experimental: VK0214 Active 40mg
40mg QD
Drug: VK0214
API in capsule

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs)
Time Frame: 28 days
Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN
28 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluate the Pharmacokinetics of VK0214
Time Frame: 28 days
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)
28 days

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluate plasma VLCFAs changes
Time Frame: 28 days
Evaluate the efficacy of VK0214 on lowering plasma levels of VLCFAs in subjects with AMN
28 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Viking Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 22, 2021

Primary Completion (Actual)

October 9, 2024

Study Completion (Actual)

November 8, 2024

Study Registration Dates

First Submitted

June 28, 2021

First Submitted That Met QC Criteria

July 21, 2021

First Posted (Actual)

July 22, 2021

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 13, 2025

Last Verified

February 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VK0214-102

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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