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

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (Very long chain fatty acid (VLCFA) levels in plasma and erythrocytes will be measured after 14 days of 50 mcg sobetirome, and again after 14 days of 100 mcg sobetirome dosing.



Original Primary Outcome: VLCFA levels [ Time Frame: Day 14 and Day 28 ]

Very long chain fatty acid (VLCFA) levels in plasma and erythrocytes will be measured after 14 and 28 days of once daily sobetirome dosing


Current Secondary Outcome:

  • Evidence of changes in thyroid function from baseline confirmed by measured changes in TSH and/or free T4 [ Time Frame: Day 14 and 28 of sobetirome dosing ]
    Thyroid function will be assessed my measurement of TSH and free T4 following 14 days of 50 mcg sobetirome, and again following 14 days of 100 mcg sobetirome dosing.
  • Number of participants with adverse events from baseline [ Time Frame: Every 7 days to outcome visit day and again at end of study visit day ]
    Adverse events will be assessed by physical examination and ECG
  • Peak Plasma Concentration (Cmax) of Sobetirome [ Time Frame: Day 1 ]
    A pharmacokinetic analysis to assess sobetirome exposure in X-ALD subjects.


Original Secondary Outcome:

  • Safety Labs [ Time Frame: Day 14 and 28 ]
    Safety labs will include serum chemistry, free fatty acid profile, hematology, urinalysis, and thyroid function.
  • Number of participants with adverse events [ Time Frame: Day 7, 14, 21 and 28 ]
    Subjects will be monitored with ECGs, vital signs, physical exams and assessment of adverse events.
  • Peak Plasma Concentration (Cmax) of Sobetirome [ Time Frame: Day 1 ]
    A pharmacokinetic analysis to assess sobetirome exposure in X-ALD subjects.


Information By: Oregon Health and Science University

Dates:
Date Received: February 6, 2013
Date Started: April 2013
Date Completion:
Last Updated: February 12, 2014
Last Verified: February 2014