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

Sponsor
Thomas S. Scanlan (Other)
Overall Status
Withdrawn
CT.gov ID
NCT01787578
Collaborator
(none)
0
Enrollment
1
Location
1
Arm
10.1
Duration (Months)
0
Patients Per Site Per Month

Study Details

Study Description

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
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.

Study Design

Study Type:
Interventional
Actual Enrollment :
0 participants
Allocation:
N/A
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 :
Apr 1, 2013
Anticipated Primary Completion Date :
Sep 1, 2013
Actual Study Completion Date :
Feb 1, 2014

Arms and Interventions

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Change from Baseline in very long chain fatty acid (VLCFA) levels [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 [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 [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 [Day 1]

      A pharmacokinetic analysis to assess sobetirome exposure in X-ALD subjects.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 64 Years
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Oregon Health & Science University Portland Oregon United States 97239

    Sponsors and Collaborators

    • Thomas S. Scanlan

    Investigators

    • Principal Investigator: David Koeller, MD, Oregon Health and Science University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Thomas S. Scanlan, Professor of Physiology & Pharmacology, Oregon Health and Science University
    ClinicalTrials.gov Identifier:
    NCT01787578
    Other Study ID Numbers:
    • Sobetirome-CLIN-006
    • CTSA grant (UL1TR000128)
    First Posted:
    Feb 8, 2013
    Last Update Posted:
    Feb 14, 2014
    Last Verified:
    Feb 1, 2014
    Keywords provided by Thomas S. Scanlan, Professor of Physiology & Pharmacology, Oregon Health and Science University
    X-linked adrenoleukodystrophy
    adrenomyeloneuropathy
    sobetirome
    thyroid
    thyromimetic
    Additional relevant MeSH terms:
    Adrenoleukodystrophy

    Study Results

    No Results Posted as of Feb 14, 2014