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

NCT01787578 | Withdrawn Phase 1 DMC

• 2 other identifiers: Sobetirome-CLIN-006CTSA grant (UL1TR000128)
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

X-Linked Adrenoleukodystrophy; Adrenomyeloneuropathy

Keywords

X-linked adrenoleukodystrophy; adrenomyeloneuropathy; sobetirome; thyroid; thyromimetic

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (3)

• 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

• Number of participants with adverse events from baseline

  Every 7 days to outcome visit day and again at end of study visit day

• Peak Plasma Concentration (Cmax) of Sobetirome

  Day 1

Study Arms (1)

Sobetirome

EXPERIMENTAL

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

Interventions

Sobetirome DRUG

50 mcg or 100 mcg once-daily oral

Also known as: GC-1, QRX-431

Sobetirome

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Thomas S. Scanlan: lead

Study Sites (1)

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Adrenoleukodystrophy; Thyroid Diseases

Interventions

GC 1 compound

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• David Koeller, MD

  Oregon Health and Science University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Physiology & Pharmacology

Study Record Dates

• First Submitted: February 6, 2013
• First Posted: February 8, 2013
• Study Start: April 1, 2013
• Primary Completion: September 1, 2013
• Study Completion: February 1, 2014
• Last Updated: February 14, 2014

Record last verified: 2014-02

Locations

US (1)