Overview

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

Status:
Recruiting
Trial end date:
2022-09-30
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Viking Therapeutics, Inc.
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 TRĪ² 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