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

Last updated: February 13, 2025
Sponsor: Viking Therapeutics, Inc.
Overall Status: Completed

Phase

1

Condition

Bone Marrow Disorder

Treatment

Placebo

VK0214

Clinical Study ID

NCT04973657
VK0214-102
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Provide a personally signed and dated informed consent document indicating that thesubject has been informed of all pertinent aspects of the study and is willing andable to participate.

  • Have diagnosis of X-linked adrenoleukodystrophy (X-ALD) by genetic testing andelevated VLCFAs.

  • Present clinical features of AMN, or adrenal insufficiency.

  • Subjects must be 18 years of age and older.

Exclusion

Exclusion Criteria:

  • Have the cerebral form of X-ALD, based on medical records, or based on MRI performedwithin the last 12 months or MRI at screening

  • Have been treated with any drug targeting TRβ or with any drug being tested astherapy 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 oroncological that in the opinion of the investigator would pose a significant riskfor the subject

  • Subject has untreated primary adrenal insufficiency based on morning plasma cortisoland ACTH at screening

  • Lorenzo's Oil. Currently taking Lorenzo's Oil or erucic acid containing product, orother lipid lowering agent known to effect VLCFA levels

Study Design

Total Participants: 24
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1
Study Start date:
June 22, 2021
Estimated Completion Date:
November 08, 2024

Study 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.

Connect with a study center

  • Viking Clinical Site 214

    Bordeaux, 33076
    France

    Site Not Available

  • Viking Clinical Site 215

    Montpellier, 34295
    France

    Site Not Available

  • Viking Clinical Site 209

    Leipzig, 04103
    Germany

    Site Not Available

  • Viking Clinical Site 210

    Milano, 20133
    Italy

    Site Not Available

  • Viking Clinical Site 212

    London, WC1N3BG
    United Kingdom

    Site Not Available

  • Viking Clinical Site 203

    Palo Alto, California 94303
    United States

    Site Not Available

  • Viking Clinical Site 207

    Gainesville, Florida 32610
    United States

    Site Not Available

  • Viking Clinical Site 201

    Baltimore, Maryland 21205
    United States

    Site Not Available

  • Viking Clinical Site 206

    New York, New York 10021
    United States

    Site Not Available

  • Viking Clinical Site 205

    Seattle, Washington 98195
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.