Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01787578
Recruitment Status : Withdrawn (revisions to original study protocol underway)
First Posted : February 8, 2013
Last Update Posted : February 14, 2014
Information provided by (Responsible Party):
Thomas S. Scanlan, Oregon Health and Science University

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.

Condition or disease Intervention/treatment Phase
X-Linked Adrenoleukodystrophy Adrenomyeloneuropathy Drug: Sobetirome Phase 1

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Arm Intervention/treatment
Experimental: Sobetirome
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.
Drug: Sobetirome
50 mcg or 100 mcg once-daily oral
Other Names:
  • GC-1
  • QRX-431

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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

  3. Peak Plasma Concentration (Cmax) of Sobetirome [ Time Frame: Day 1 ]
    A pharmacokinetic analysis to assess sobetirome exposure in X-ALD subjects.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01787578

Layout table for location information
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Thomas S. Scanlan
Layout table for investigator information
Principal Investigator: David Koeller, MD Oregon Health and Science University

Layout table for additonal information
Responsible Party: Thomas S. Scanlan, Professor of Physiology & Pharmacology, Oregon Health and Science University Identifier: NCT01787578     History of Changes
Other Study ID Numbers: Sobetirome-CLIN-006
CTSA grant (UL1TR000128) ( Other Grant/Funding Number: Oregon Clinical & Translational Research Institute )
First Posted: February 8, 2013    Key Record Dates
Last Update Posted: February 14, 2014
Last Verified: February 2014
Keywords provided by Thomas S. Scanlan, Oregon Health and Science University:
X-linked adrenoleukodystrophy
Additional relevant MeSH terms:
Layout table for MeSH terms
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases