New clinical trial for adrenoleukodystrophy : effect of sobetirome on very long chain fatty acids



Written on Thursday 11 April 2013

IMPORTANT : This study has been withdrawn prior to enrollment. Revisions to original study protocol are underway.

Status: Active; Recruiting patients
Experimental drug: Sobetirome, a selective thyroid hormone analog
Objective: To assess the safety, tolerance, pharmacokinetics, and pharmacodynamics of sobetirome in adult male ALD patients.

Investigators
Dr. David M. Koeller, Oregon Health & Science University, Portland, Oregon, United States
Pr. Thomas S. Scanlan, Oregon Health & Science University, Portland, Oregon, United States

Start date: April 2013
End of the study: October 2013 (estimated)
Location: Oregon Health & Science University, Portland, Oregon, United States
Funding: OHSU
Reference: NCT01787578

Patients
10 male patients with adrenoleukodystrophy (ALD) between 18 and 65 years old.

Treatment
Subjects will receive oral doses of sobetirome. All subjects will start with a 50 microgram dose, once-daily for 14 days. If this dose proves safe and well tolerated, subjects will receive a 100 microgram dose once-daily for an additional 14 days.

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  very long chain fatty acids (VLCFAs). Subjects will receive an oral dose of 50 microgram sobetirome once daily for 14 days beginning on Day 1. 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 microgram 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.

Inclusion criteria

  • males 18-65 years old
  • ALD diagnosed by either elevated VLCFAs or DNA testing

Exclusion criteria

  • abnormal laboratory test results (except VLCFAs) at screening visit
  • history of coronary artery disease
  • use of thyroid hormone therapy
  • abnormal thyroid function test at screening visit
  • untreated adrenal insufficiency
  • currently taking Lorenzo’s Oil or other VLCFAs lowering agent

Source: ClinicalTrials.gov

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