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

NCT ID: NCT01787578

Last Updated: 2014-02-14

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-04-30
• Study Completion Date: 2014-02-28

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.

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.

Conditions

X-Linked Adrenoleukodystrophy; Adrenomyeloneuropathy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Sobetirome

Intervention Type: DRUG

50 mcg or 100 mcg once-daily oral

Interventions

Sobetirome

50 mcg or 100 mcg once-daily oral

Intervention Type: DRUG

Other Intervention Names

GC-1; QRX-431

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 64 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Thomas S. Scanlan

OTHER

Sponsor Role: lead

Responsible Party

Thomas S. Scanlan

Professor of Physiology & Pharmacology

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

David Koeller, MD

Role: PRINCIPAL_INVESTIGATOR

Oregon Health and Science University

Locations

Oregon Health & Science University

Portland, Oregon, United States

Countries

United States

Other Identifiers

CTSA grant (UL1TR000128)

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

Sobetirome-CLIN-006

Identifier Type: -

Identifier Source: org_study_id