Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
|ClinicalTrials.gov Identifier: NCT01787578|
Recruitment Status : Withdrawn (revisions to original study protocol underway)
First Posted : February 8, 2013
Last Update Posted : February 14, 2014
|Condition or disease||Intervention/treatment||Phase|
|X-Linked Adrenoleukodystrophy Adrenomyeloneuropathy||Drug: Sobetirome||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (X-ALD)|
|Study Start Date :||April 2013|
|Estimated Primary Completion Date :||September 2013|
|Actual Study Completion Date :||February 2014|
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.
50 mcg or 100 mcg once-daily oral
- Change from Baseline in very long chain fatty acid (VLCFA) levels [ Time Frame: Day 14 and Day 28 of sobetirome dosing ]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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01787578
|United States, Oregon|
|Oregon Health & Science University|
|Portland, Oregon, United States, 97239|
|Principal Investigator:||David Koeller, MD||Oregon Health and Science University|