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

NCT04973657 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: VK0214-102
• Study Type: interventional
• Target: 24
• Locations: 5 countries
• Sites: 9

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.

Conditions

Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

Keywords

AMN, XALD

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Evaluate the Pharmacokinetics of VK0214

  28 days

Other Outcomes (1)

• Evaluate plasma VLCFAs changes

  28 days

Study Arms (3)

VK0214 Active 20mg

EXPERIMENTAL

20mg QD

Drug: VK0214

VK0214 Active 40mg

EXPERIMENTAL

40mg QD

Drug: VK0214

Placebo

PLACEBO COMPARATOR

Placebo QD

Other: Placebo

Interventions

VK0214 DRUG

API in capsule

VK0214 Active 20mg; VK0214 Active 40mg

Placebo OTHER

API in capsule

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Viking Therapeutics, Inc.: lead

Study Sites (9)

Viking Clinical Site 203

Palo Alto, California, 94303, United States

Location

Viking Clinical Site 207

Gainesville, Florida, 32610, United States

Location

Viking Clinical Site 201

Baltimore, Maryland, 21205, United States

Location

Viking Clinical Site 205

Seattle, Washington, 98195, United States

Location

Viking Clinical Site 214

Bordeaux, 33076, France

Location

Viking Clinical Site 215

Montpellier, 34295, France

Location

Viking Clinical Site 209

Leipzig, 04103, Germany

Location

Viking Clinical Site 210

Milan, 20133, Italy

Location

Viking Clinical Site 212

London, WC1N3BG, United Kingdom

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2021
• First Posted: July 22, 2021
• Study Start: June 22, 2021
• Primary Completion: October 9, 2024
• Study Completion: November 8, 2024
• Last Updated: February 17, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1); FR (2); US (4); IT (1); GB (1)