Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)Phase 1NCT04973657
A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN
A Phase 1 study testing VK0214 in people with Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD), run by Viking Therapeutics, Inc. at 9 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
- What is being tested?
- VK0214
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 22 Jun 2021to 8 Nov 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
United States4 sites
Viking Clinical Site 201
Baltimore, Maryland, 21205
Viking Clinical Site 207
Gainesville, Florida, 32610
Viking Clinical Site 203
Palo Alto, California, 94303
Viking Clinical Site 205
Seattle, Washington, 98195
France2 sites
Viking Clinical Site 214
Bordeaux, 33076
Viking Clinical Site 215
Montpellier, 34295
About this study
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)
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.
Read the 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.
- Being tested
- VK0214, Placebo
- Keywords
- AMN, XALD
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VK0214Drug
API in capsule
PlaceboOther
API in capsule
| Study group | Role | What they receive |
|---|---|---|
| VK0214 Active 20mg | Experimental | 20mg QD Receives: Drug: VK0214 |
| VK0214 Active 40mg | Experimental | 40mg QD Receives: Drug: VK0214 |
| Placebo | Placebo comparator | Placebo QD Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 28 days
Secondary outcomes (1)
Evaluate the Pharmacokinetics of VK0214
Pharmacokinetic profile of VK0214 by measuring Peak Plasma Concentration (Cmax)
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2021
- Study start22 Jun 2021
- Primary completion9 Oct 2024
- Study completion8 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Viking Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04973657
- Sponsor's protocol ID
- VK0214-102
- Status last verified
- Feb 2025
- First submitted
- 28 Jun 2021
- Last updated
- 17 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04973657
- Acronym / sponsor ID
- VK0214-102
- Keywords
- AMN, XALD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Viking Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Jun 2021 (actual)
- Primary completion
- 9 Oct 2024 (actual)
- Study completion
- 8 Nov 2024 (actual)
- First posted
- 22 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| VK0214 Active 20mg | Experimental | Drug: VK021420mg QD |
| VK0214 Active 40mg | Experimental | Drug: VK021440mg QD |
| Placebo | Placebo comparator | Other: PlaceboPlacebo QD |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VK0214API in capsule | Drug | VK0214 Active 20mg, VK0214 Active 40mg |
| PlaceboAPI in capsule | Other | Placebo |
Primary outcome
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
1 secondary outcome
Evaluate the Pharmacokinetics of VK0214
Time frame: 28 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2025.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VK0214 in people with Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD), run by Viking Therapeutics, Inc. at 9 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, France, Germany, Italy, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs) (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2025.