Brief Title
Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
Official Title
A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (X-ALD)
Brief Summary
The purpose of this study is to assess the safety, tolerance, pharmacokinetics, and
pharmacodynamics of sobetirome, a selective thyroid hormone analog, in adult male X-ALD
patients.
Detailed Description
Subjects will have a screening visit within 6 weeks prior to the Baseline visit. At Baseline
visit blood will be drawn and to establish baseline values for plasma and red blood cell
(RBC) very long chain fatty acids (VLCFA; C22, C24, and C26). Subjects will receive an oral
dose of 50 mcg sobetirome once daily for 14 days beginning on Day 1. Subjects will be kept in
the clinic on Day 1 for 16 hours following their initial dose of sobetirome for repeat blood
sampling for pharmacokinetic analysis. Subjects will return to the clinic on days 7, 15, 21
and 28 for blood collection for VLCFA measurements. On day 15, after safety assessment,
subjects will receive an increased dose of 100 mcg and this dose will be continued once daily
through Day 28. Subjects will continue to return to the clinic weekly for blood and urine
collection and safety assessments. Subjects will return to the clinic on day 42 for an End of
Study visit that will involve a final measurement of VLCFA and blood and urine safety labs to
check for reversibility. Safety labs will include serum chemistry, free fatty acid profile,
hematology, urinalysis, and thyroid function. Subjects will be monitored with ECGs, vital
signs, physical exams and assessment of adverse events.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Change from Baseline in very long chain fatty acid (VLCFA) levels
Secondary Outcome
Evidence of changes in thyroid function from baseline confirmed by measured changes in TSH and/or free T4
Condition
X-Linked Adrenoleukodystrophy
Intervention
Sobetirome
Study Arms / Comparison Groups
Sobetirome
Description: Subjects will receive oral doses of sobetirome. All subjects will start with a 50 mcg dose, once-daily for 14 days. If this dose proves safe and well tolerated, subjects will receive a 100 mcg dose once-daily for an additional 14 days.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
0
Start Date
April 2013
Completion Date
February 2014
Primary Completion Date
September 2013
Eligibility Criteria
Inclusion Criteria:
- males 18-65 years old
- X-ALD diagnosis by either elevated VLCFAs or DNA testing
- must sign informed consent and agree to complete required clinic visits.
Exclusion Criteria:
- female gender
- abnormal laboratory test results (except VLCFA) at screening visit
- history of coronary artery disease
- use of triiodothyronine therapy
- abnormal thyroid function test at screening visit
- untreated adrenal insufficiency
- currently taking Lorenzo's Oil or other VLCFA lowering agent
- participation in investigational drug study within 30 days
Gender
Male
Ages
18 Years - 64 Years
Accepts Healthy Volunteers
No
Contacts
David Koeller, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT01787578
Organization ID
Sobetirome-CLIN-006
Secondary IDs
CTSA grant (UL1TR000128)
Responsible Party
Sponsor-Investigator
Study Sponsor
Thomas S. Scanlan
Study Sponsor
David Koeller, MD, Principal Investigator, Oregon Health and Science University
Verification Date
February 2014