Marfan SyndromePhase 3NCT01361087
Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome
A Phase 3 study testing Blood draw in people with Marfan Syndrome, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and… Read more about this study
- Who is it for?
- Ages 6 months to 24 years
- Conditions the study is for
- Marfan Syndrome
- What is being tested?
- Blood draw
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2011to Dec 2014
- Phase
- Phase 3
- 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 6 months to 24 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Individual with Marfan syndrome consented in to the Main Atenolol Vs. Losartan NIH study.
Who cannot join
- Subjects in the main PHN Marfan trial who have not achieved the maintenance drug dosing or who have stopped taking study drug.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Memorial Hospital Chicago
Chicago, Illinois, 60614
About this study
Official title: Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and heart complications such as mitral valve prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I, normally an important component of elastic fibers it has been shown that the Marfan syndrome phenotype can be relieved by addition of a TGF-β antagonist in affected mice.
Read the detailed description
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and heart complications such as mitral valve prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I, normally an important component of elastic fibers it has been shown that the Marfan syndrome phenotype can be relieved by addition of a TGF-β antagonist in affected mice. This suggest that while the symptoms of Marfan syndrome may seem consistent with a connective tissue disorder, the mechanism is more likely related to reduced sequestration of TGF-β by fibrillin.
- Conditions
- Marfan Syndrome
- Being tested
- Blood draw
- Keywords
- TGF-B blood levels Atenolol vs, Losartan Study, NIH, NIHLB and NMF
- Medical subject headings
- Marfan Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood drawOther
This study includes one blood draw to measure circulating blood levels of TGF-B.
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan.
Measured: 1 year
Secondary outcomes (1)
To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups.
These clinical outcomes may be a change in aortic root Z-score, final aortic root dimension, final aortic root Z-score and other clinical outcomes in the main Marfan trial.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2011 (estimated)
- Study startApr 2011
- Primary completionDec 2014
Why it stopped: No outside recruitment of subjects from Main Atenolol VS Losartan NIH study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Marfan SyndromeStudies 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 3
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01361087
- Sponsor's protocol ID
- IRB # 2011-14507
- Status last verified
- Feb 2016
- First submitted
- 25 May 2011
- Last updated
- 5 Feb 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With
- ClinicalTrials.gov ID
- NCT01361087
- Acronym / sponsor ID
- IRB # 2011-14507
- Conditions
- Marfan Syndrome
- Keywords
- TGF-B blood levels Atenolol vs, Losartan Study, NIH, NIHLB and NMF
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No outside recruitment of subjects from Main Atenolol VS Losartan NIH study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- Johns Hopkins University
- Study start
- Apr 2011
- Primary completion
- Dec 2014 (actual)
- First posted
- 26 May 2011 (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.
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and heart complications such as mitral valve prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I, normally an important component of elastic fibers it has been shown that the Marfan syndrome phenotype can be relieved by addition of a TGF-β antagonist in affected mice.
Detailed description
Transforming Growth Factor Beta (TGF-β) is a protein that controls proliferation, cellular differentiation, and other functions in most cells. TGF-β levels play a major role in the pathogenesis of Marfan syndrome, a disease characterized by disproportionate height, long extremities, lens dislocation in the eyes and heart complications such as mitral valve prolapse and aortic enlargement increasing the likelihood of aortic dissection. While the underlying defect in Marfan syndrome is faulty synthesis of the glycoprotein fibrillin I, normally an important component of elastic fibers it has been shown that the Marfan syndrome phenotype can be relieved by addition of a TGF-β antagonist in affected mice. This suggest that while the symptoms of Marfan syndrome may seem consistent with a connective tissue disorder, the mechanism is more likely related to reduced sequestration of TGF-β by fibrillin.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 6 months to 24 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Individual with Marfan syndrome consented in to the Main Atenolol Vs. Losartan NIH study.
Key exclusion criteria (1 listed)
- Subjects in the main PHN Marfan trial who have not achieved the maintenance drug dosing or who have stopped taking study drug.
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 |
|---|---|---|
| Blood drawThis study includes one blood draw to measure circulating blood levels of TGF-B. | Other | – |
Primary outcome
To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan.
Time frame: 1 year
1 secondary outcome
To determine if circulating levels of TGF-β correlate with clinical outcomes within a treatment group or independent treatment groups.
Time frame: 1 year
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 5 Feb 2016.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Blood draw in people with Marfan Syndrome, run by Ann & Robert H Lurie Children's Hospital of Chicago 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 1 inclusion and 1 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 5 Feb 2016. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
To determine if circulating levels of TGF-β correlate with treatment arms: Atenolol vs. Losartan. (time frame: 1 year). 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 5 Feb 2016.