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

Sponsor

Viking Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04973657

Collaborator

(none)

Enrollment

Locations

Arms

20.2

Anticipated Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)	Drug: VK0214 Other: Placebo	Phase 1

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 Design

Study Type:

Interventional

Anticipated Enrollment :

36 participants

Allocation:

Randomized

Intervention Model:

Sequential Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Other

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)

Actual Study Start Date :

Jun 22, 2021

Anticipated Primary Completion Date :

Nov 30, 2022

Anticipated Study Completion Date :

Feb 28, 2023

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: VK0214 Active 20mg

20mg QD

Drug: VK0214

API in capsule

Experimental: VK0214 Active 40mg

40mg QD

Drug: VK0214

API in capsule

Placebo Comparator: Placebo

Placebo QD

Other: Placebo

API in capsule

Outcome Measures

Primary Outcome Measures

Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs) [28 days]
Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN

Secondary Outcome Measures

Evaluate the Pharmacokinetics of VK0214 [28 days]
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)

Other Outcome Measures

Evaluate plasma VLCFAs changes [28 days]
Evaluate the efficacy of VK0214 on lowering plasma levels of VLCFAs in subjects with AMN

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Viking Clinical Site 203	Palo Alto	California	United States	94303
2	Viking Clinical Site 207	Gainesville	Florida	United States	32610
3	Viking Clinical Site 201	Baltimore	Maryland	United States	21205
4	Viking Clinical Site 205	Seattle	Washington	United States	98195

Sponsors and Collaborators

Viking Therapeutics, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Viking Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT04973657

Other Study ID Numbers:

VK0214-102

First Posted:

Jul 22, 2021

Last Update Posted:

Jul 20, 2022

Last Verified:

Jul 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Viking Therapeutics, Inc.

AMN, XALD

Additional relevant MeSH terms:

Adrenoleukodystrophy

Study Results

No Results Posted as of Jul 20, 2022