X-Linked AdrenoleukodystrophyPhase 1NCT01787578
Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
A Phase 1 study testing Sobetirome in people with X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy, run by Thomas S. Scanlan at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 to 64 years, men only
- Conditions the study is for
- X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
- What is being tested?
- Sobetirome
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2013to Feb 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (X-ALD)
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.
Read the 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.
- Being tested
- Sobetirome
- Keywords
- X-linked adrenoleukodystrophy, adrenomyeloneuropathy, sobetirome, thyroid, thyromimetic
- Medical subject headings
- Adrenoleukodystrophy, Thyroid Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SobetiromeDrug
Also called GC-1, QRX-431
50 mcg or 100 mcg once-daily oral
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Sobetirome |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
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.
Measured: Day 14 and 28 of sobetirome dosing
Number of participants with adverse events from baseline
Adverse events will be assessed by physical examination and ECG
Measured: Every 7 days to outcome visit day and again at end of study visit day
Peak Plasma Concentration (Cmax) of Sobetirome
A pharmacokinetic analysis to assess sobetirome exposure in X-ALD subjects.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2013 (estimated)
- Study startApr 2013
- Primary completionSep 2013 (estimated)
- Study completionFeb 2014
Why it stopped: revisions to original study protocol underway
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked AdrenoleukodystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Thomas S. Scanlan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Thomas S. Scanlan, Oregon Health and Science University
- Investigators
- David Koeller, MD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01787578
- Sponsor's protocol ID
- Sobetirome-CLIN-006
- Other IDs
- CTSA grant (UL1TR000128)
- Status last verified
- Feb 2014
- First submitted
- 6 Feb 2013
- Last updated
- 14 Feb 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (X-ALD)
- ClinicalTrials.gov ID
- NCT01787578
- Acronym / sponsor ID
- Sobetirome-CLIN-006
- Keywords
- X-linked adrenoleukodystrophy, adrenomyeloneuropathy, sobetirome, thyroid, thyromimetic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: revisions to original study protocol underway
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Thomas S. ScanlanUniversity, hospital or other
- Study start
- Apr 2013
- Primary completion
- Sep 2013 (estimated)
- Study completion
- Feb 2014 (actual)
- First posted
- 8 Feb 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sobetirome | Experimental | Drug: SobetiromeSubjects 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. |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SobetiromeAlso: GC-1, QRX-43150 mcg or 100 mcg once-daily oral | Drug | Sobetirome |
Primary outcome
Change from Baseline in very long chain fatty acid (VLCFA) levels
Time frame: 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.
3 secondary outcomes
Evidence of changes in thyroid function from baseline confirmed by measured changes in TSH and/or free T4
Time frame: Day 14 and 28 of sobetirome dosing
Number of participants with adverse events from baseline
Time frame: Every 7 days to outcome visit day and again at end of study visit day
Peak Plasma Concentration (Cmax) of Sobetirome
Time frame: Day 1
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2014.
Related trials for X-Linked Adrenoleukodystrophy
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
Not yet recruiting
IT and IV Lentiviral Gene Therapy for X-ALD
X-linked Adrenoleukodystrophy
Recruiting
Home Exercise for Individuals With Neurodegenerative Disease
Neurodegenerative Diseases, Leukodystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Koeller, MD principal investigator
Oregon Health and Science University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Sobetirome in people with X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy, run by Thomas S. Scanlan at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 14 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in very long chain fatty acid (VLCFA) levels (time frame: Day 14 and Day 28 of sobetirome dosing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Feb 2014.