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

X-Linked Adrenoleukodystrophy Clinical Trial

Official title:

A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (X-ALD)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01787578
• Other study ID #: Sobetirome-CLIN-006
• Secondary ID: CTSA grant (UL1T
• Status: Withdrawn
• Phase: Phase 1
• First received: February 6, 2013
• Last updated: February 12, 2014
• Start date: April 2013
• Est. completion date: February 2014

Study information

• Verified date: February 2014
• Source: Oregon Health and Science University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: February 2014
• Est. primary completion date: September 2013
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 64 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Adrenomyeloneuropathy
• X-Linked Adrenoleukodystrophy

Intervention

Drug:
• Sobetirome
50 mcg or 100 mcg once-daily oral

Locations

Country	Name	City	State
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Thomas S. Scanlan

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in very long chain fatty acid (VLCFA) levels	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.	Day 14 and Day 28 of sobetirome dosing	No
Secondary	Evidence of changes in thyroid function from baseline confirmed by measured changes in TSH and/or free T4	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.	Day 14 and 28 of sobetirome dosing	Yes
Secondary	Number of participants with adverse events from baseline	Adverse events will be assessed by physical examination and ECG	Every 7 days to outcome visit day and again at end of study visit day	Yes
Secondary	Peak Plasma Concentration (Cmax) of Sobetirome	A pharmacokinetic analysis to assess sobetirome exposure in X-ALD subjects.	Day 1	No