Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

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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