Single Ventricle Fontan PalliationPhase 4NCT01971580
The Effects of Ambrisentan on Exercise Capacity in Fontan Patients
A Phase 4 study testing Ambrisentan in people with Single Ventricle Fontan Palliation, run by Washington University School of Medicine at 1 site in United States.
Trial summary
Hypothesis: Patients with a Fontan type palliation are limited by preload, or the rate at which blood returns to the heart after passively traversing the pulmonary capillary bed. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Single Ventricle Fontan Palliation
- What is being tested?
- Ambrisentan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2012to May 2015
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients over 18 years of age followed at the Center for Adults with Congenital Heart Disease at Washington University School of Medicine or at Saint Louis Childrens Hospital who have had a Fontan-type of palliation with single ventricular physiology.
- Patients must not within the last year have been treated with an endothelin receptor antagonist, phosphodiesterase 5 antagonist, or prostacyclin analogue.
- Female patients must not be pregnant, and during the study period must be using effective contraception (including barrier method with spermicide, intrauterine device, implanon, oral contraceptives) if fertile.
- In the physicians opinion patients must be able to complete a VO2 max test to achieve an RER of >1.0
- In the physicians opinion patients must be socially and intellectually able to adhere to the treatment regimen and follow-up
- Patients must be intellectually able to reliably complete an SF-36 questionnaire on quality of life.
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- Patients must be clinically stable for at least 3 months prior to enrolment.
- Patients must not have had surgery within 6 months prior to enrolment, and have no planned surgery for the study period.
- Patients must provide informed consent.
- -
Who cannot join
- Patients under 18 years of age.
- Patients who are pregnant, nursing, or who in the physician's opinion are likely to become pregnant.
- Patients who are clinically unstable.
- Patients who have previously been treated with either an endothelin receptor antagonist and stopped due to adverse side effects.
- Patients with an AST or ALT of >3 x normal.
- Patients having any known contraindication to the initiation of ambrisentan.
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- Patients with a calculated creatinine clearance of <60ml/min
- Patients with a serum hemoglobin concentration of <10mg/dl
- Patients with a poorly controlled cardiac arrhythmia
- Patients who are unable to provide independent informed consent.
- -
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Hypothesis:
Patients with a Fontan type palliation are limited by preload, or the rate at which blood returns to the heart after passively traversing the pulmonary capillary bed. By decreasing pulmonary vascular resistance using an endothelin receptor antagonist, both ventricular filling pressures and volumes will increase with a simultaneous decrease in systemic impedance to flow and decrease in central venous pressures, leading to an improved capacity to increase cardiac output, and thereby an improvement in patient functional status.
Patients who are candidates for the study will be randomized to a double-blind single crossover study. Therapy with either ambrisentan or placebo will be continued for 12 weeks, with a 2 week washout period between treatment periods. Subjects will be subjected to a VO2 max test to evaluate exercise capacity at enrolment, and on the last day of each treatment period. As a component of the VO2 max testing the patient's VE, VCO2, VE/VCO2 slope, ventilatory anaerobic threshold (VAT), peak work, efficiency, and other physiologic parameters as typically obtained from cardiopulmonary testing will be assessed. In addition, each patient will be asked to complete an SF-36 quality of life questionnaire at enrollment, and on the last day of each study period.
- Conditions
- Single Ventricle Fontan Palliation
- Being tested
- Ambrisentan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AmbrisentanDrug
Also called Letaris
| Study group | Role | What they receive |
|---|---|---|
| Ambrisentan first, Placebo second | Other | These patients will be randomized to have ambrisentan during the first study period, and placebo during the second study period. Receives: Drug: Ambrisentan |
| Placebo first, Ambrisentan second | Other | These patients will be randomized to have placebo during the first study period, and ambrisentan during the second study period. Receives: Drug: Ambrisentan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in VO2 Max
Measured: Baseline compared to 12 weeks therapy with either ambrisentan or placebo
Secondary outcomes (1)
Quality of Life
Change in Rand 36-item Short Form (SF-36) physical function score (0-100). On the Rand SF-36 questionnaire, a score closer to 100 (higher) suggests lesser disability, while a score closer to 0 (lower) suggests more disability.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2013 (estimated)
- Study startAug 2012
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Single Ventricle Fontan PalliationStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ari Cedars, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01971580
- Sponsor's protocol ID
- IN-US-300-D076
- Status last verified
- May 2018
- First submitted
- 23 Oct 2013
- Last updated
- 7 May 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of Ambrisentan on Exercise Capacity in Fontan Patients
- ClinicalTrials.gov ID
- NCT01971580
- Acronym / sponsor ID
- IN-US-300-D076
- Conditions
- Single Ventricle Fontan Palliation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- Aug 2012
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 29 Oct 2013 (estimated)
- Results
- Posted 7 May 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hypothesis:
Patients with a Fontan type palliation are limited by preload, or the rate at which blood returns to the heart after passively traversing the pulmonary capillary bed. By decreasing pulmonary vascular resistance using an endothelin receptor antagonist, both ventricular filling pressures and volumes will increase with a simultaneous decrease in systemic impedance to flow and decrease in central venous pressures, leading to an improved capacity to increase cardiac output, and thereby an improvement in patient functional status.
Patients who are candidates for the study will be randomized to a double-blind single crossover study. Therapy with either ambrisentan or placebo will be continued for 12 weeks, with a 2 week washout period between treatment periods. Subjects will be subjected to a VO2 max test to evaluate exercise capacity at enrolment, and on the last day of each treatment period. As a component of the VO2 max testing the patient's VE, VCO2, VE/VCO2 slope, ventilatory anaerobic threshold (VAT), peak work, efficiency, and other physiologic parameters as typically obtained from cardiopulmonary testing will be assessed. In addition, each patient will be asked to complete an SF-36 quality of life questionnaire at enrollment, and on the last day of each study period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ambrisentan first, Placebo second | Other | Drug: AmbrisentanThese patients will be randomized to have ambrisentan during the first study period, and placebo during the second study period. |
| Placebo first, Ambrisentan second | Other | Drug: AmbrisentanThese patients will be randomized to have placebo during the first study period, and ambrisentan during the second study period. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Patients over 18 years of age followed at the Center for Adults with Congenital Heart Disease at Washington University School of Medicine or at Saint Louis Childrens Hospital who have had a Fontan-type of palliation with single ventricular physiology.
- Patients must not within the last year have been treated with an endothelin receptor antagonist, phosphodiesterase 5 antagonist, or prostacyclin analogue.
- Female patients must not be pregnant, and during the study period must be using effective contraception (including barrier method with spermicide, intrauterine device, implanon, oral contraceptives) if fertile.
- In the physicians opinion patients must be able to complete a VO2 max test to achieve an RER of >1.0
- In the physicians opinion patients must be socially and intellectually able to adhere to the treatment regimen and follow-up
Key exclusion criteria (11 listed)
- Patients under 18 years of age.
- Patients who are pregnant, nursing, or who in the physician's opinion are likely to become pregnant.
- Patients who are clinically unstable.
- Patients who have previously been treated with either an endothelin receptor antagonist and stopped due to adverse side effects.
- Patients with an AST or ALT of >3 x normal.
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 |
|---|---|---|
| AmbrisentanAlso: Letaris | Drug | Ambrisentan first, Placebo second, Placebo first, Ambrisentan second |
Primary outcome
Change in VO2 Max
Time frame: Baseline compared to 12 weeks therapy with either ambrisentan or placebo
1 secondary outcome
Quality of Life
Time frame: 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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 4 study testing Ambrisentan in people with Single Ventricle Fontan Palliation, run by Washington University School of Medicine 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 10 inclusion and 11 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: finished, as of the sponsor's last update on 7 May 2018. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 in VO2 Max (time frame: Baseline compared to 12 weeks therapy with either ambrisentan or placebo). 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 7 May 2018.