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

Primary Purpose

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

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
VK0214
Placebo
Sponsored by
Viking Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) focused on measuring AMN, XALD

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Viking Clinical Site 203Recruiting
  • Viking Clinical Site 207Recruiting
  • Viking Clinical Site 201Recruiting
  • Viking Clinical Site 205Recruiting
  • Viking Clinical Site 214Recruiting
  • Viking Clinical Site 215Recruiting
  • Viking Clinical Site 209Recruiting
  • Viking Clinical Site 210Recruiting
  • Viking Clinical Site 212

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

VK0214 Active 20mg

VK0214 Active 40mg

Placebo

Arm Description

20mg QD

40mg QD

Placebo QD

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Evaluate the Pharmacokinetics of VK0214
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)

Full Information

First Posted
June 28, 2021
Last Updated
May 18, 2023
Sponsor
Viking Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04973657
Brief Title
A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 22, 2021 (Actual)
Primary Completion Date
September 30, 2023 (Anticipated)
Study Completion Date
December 30, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Viking Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
Keywords
AMN, XALD

7. Study Design

Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
36 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VK0214 Active 20mg
Arm Type
Experimental
Arm Description
20mg QD
Arm Title
VK0214 Active 40mg
Arm Type
Experimental
Arm Description
40mg QD
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo QD
Intervention Type
Drug
Intervention Name(s)
VK0214
Intervention Description
API in capsule
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
API in capsule
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs)
Description
Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN
Time Frame
28 days
Secondary Outcome Measure Information:
Title
Evaluate the Pharmacokinetics of VK0214
Description
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)
Time Frame
28 days
Other Pre-specified Outcome Measures:
Title
Evaluate plasma VLCFAs changes
Description
Evaluate the efficacy of VK0214 on lowering plasma levels of VLCFAs in subjects with AMN
Time Frame
28 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Stephanie Herman
Phone
858-704-4694
Email
sherman@vikingtherapeutics.com
First Name & Middle Initial & Last Name or Official Title & Degree
Marianne Mancini
Phone
858-704-4674
Email
mmancini@vikingtherapeutics.com
Facility Information:
Facility Name
Viking Clinical Site 203
City
Palo Alto
State/Province
California
ZIP/Postal Code
94303
Country
United States
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 207
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 201
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 205
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 214
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 215
City
Montpellier
ZIP/Postal Code
34295
Country
France
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 209
City
Leipzig
ZIP/Postal Code
04103
Country
Germany
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 210
City
Milano
ZIP/Postal Code
20133
Country
Italy
Individual Site Status
Recruiting
Facility Name
Viking Clinical Site 212
City
London
ZIP/Postal Code
WC1N3BG
Country
United Kingdom
Individual Site Status
Not yet recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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