Congenital Heart DiseasePhase 1NCT01698567
Antithrombin III in Infants With Cardiopulmonary Bypass (CPB)
A Phase 1 study testing Antithrombin III in people with Congenital Heart Disease, run by University of Rochester at 1 site in United States.
Trial summary
The purpose of this study is to discern whether supplementation of Antithrombin III will decrease coagulation and inflammation associated with cardiopulmonary bypass in infants undergoing cardiac surgery. Read more about this study
- Who is it for?
- Ages 1 day to 180 days
- Conditions the study is for
- Congenital Heart Disease
- What is being tested?
- Antithrombin III
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2012to Mar 2016
- 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 1 day to 180 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subjects will be included if they are presenting for elective cardiac surgery using cardiopulmonary bypass and are 180 days of age or less.
Who cannot join
- Sensitivity to ATIII product (Thrombate, Grifols, Los Angeles, CA)
- Known inherited or acquired coagulation defect
- Current or prior treatment with pro-or anticoagulant medication within past 30 days (except aspirin or a single dose of heparin, e.g. for catheterization)
- Known central venous thrombosis
- Recent (30 days) transfusion with hemostatic blood products (fresh-frozen plasma, platelets, cryoprecipitate, whole blood)
- wt less than 3000g
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Antithrombin III Supplementation for Infants Undergoing Cardiac Surgery With Cardiopulmonary Bypass.
The purpose of this study is to discern whether supplementation of Antithrombin III will decrease coagulation and inflammation associated with cardiopulmonary bypass in infants undergoing cardiac surgery.
Read the detailed description
Cardiopulmonary bypass (CPB) and cardiovascular surgery initiate diffuse activation of coagulation, inflammation and fibrinolysis that often has deleterious effects on patient outcomes, including bleeding, transfusion, myocardial dysfunction, renal failure, pulmonary dysfunction, prolonged intubation, ICU and hospital length of stay, stroke and neurocognitive dysfunction, and mortality.Pediatric patients are especially at risk for hematologic derangement related to CPB. Not only are infants and children subject to the same diffuse activations of coagulation, inflammation, and fibrinolysis as adults, but their size, immaturity, and circulatory abnormalities secondary to congenital heart disease increase the risk of loss of hematologic homeostasis. Since an infant's blood volume is much smaller than that of the prime in the CPB pump, hemodilution alone produces impaired hemostasis related to thrombocytopenia and coagulation factor dilution The incidence of each of these complications is variable and depends on the diagnosis, operation, time on CPB and other factors.
Adequate anticoagulation during cardiopulmonary bypass (CPB) is essential to preserve the hemostatic system and ensure hemostasis after surgery. Heparin has long been the mainstay of anticoagulation for CPB, due to its ease of use, familiarity, and reversibility. For heparin to exert its anticoagulant effect, it must bind with an intrinsic cofactor, antithrombin III (ATIII) to inhibit enzymes of the intrinsic and final common coagulation pathways. It has been established that neonates have significantly decreased levels of ATIII relative to adults, and that this relative deficiency continues at least until 6 months of age. Children with congenital heart disease have further decreases in ATIII and other abnormalities of coagulation which may contribute to adverse outcomes. Given this ATIII deficiency, it is not surprising that heparin anticoagulation does not fully suppress coagulation during CPB. Neonates anticoagulated for CPB with heparin have ongoing activation of humoral and cellular coagulation with associated activation of inflammation and fibrinolysis. Improved anticoagulation may reduce activation of these cascades and improve outcomes. In addition, infants are at high risk for post cardiac surgery intervascular thrombosis. Baseline ATIII deficiency, and consumption of ATIII during bypass may contribute to a postoperative prothrombotic state esulting in this often fatal complication. ATIII supplementation may decrease this risk.
ATIII is available as a lyophilized product derived from pooled human plasma. Treatment with ATIII has been shown to improve the anticoagulant effects of heparin and attenuate activation of hemostasis and inflammation during adult CPB, and to decrease the incidence of thrombosis associated with central venous cannulation in children. The incidence of central venous thrombosis in infants undergoing cardiac surgery has been reported as 5.8 - 22% in neonates, with a resultant mortality of 20%.
The biologic half-life of antithrombin in healthy adult volunteers is 2.5 - 3.8 days. Pharmacokinetic data in neonates is not available, but biologic activity should certainly persist through the 2 highest risk periods:
CPB, where activation of coagulation produces activation of inflammation, both cellular and humoral, and fibrinolysis.
The early post-op period when patients typically become hypercoagulable as part of the stress response to surgery. Hypercoagulability places the patients at high risk for central line-associated thrombosis despite by heparin-containing flush solutions which are standard of care. Ensuring a near normal level of antithrombin appears to enhance the ability of the flush solutions to inhibit thrombin generation on the catheter.
ATIII has been used in infants after cardiac surgery to prophylax against central venous thrombosis, for infants with necrotizing enterocolitis (NEC), and as treatment for neonates with congenital ATIII deficiency. Neonates appear to respond as expected to ATIII supplementation, with clinical efficacy for central venous thrombosis but not NEC. No complications unique to infants were reported in any of these publications.
It is reasonable to expect that ATIII would be even more beneficial in infants less than 6 months old.
- Conditions
- Congenital Heart Disease
- Being tested
- Antithrombin III, Placebo
- Keywords
- Infants, Cardiopulmonary bypass, anticoaglution
- Medical subject headings
- Heart Defects, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Antithrombin IIIDrug
Also called Thrombate
ATIII will be dosed using the formula recommended by the manufacturer:
(goal activity - baseline activity) x weight (kg) x .714 (Assume: start with 35% activity [7], goal 120% activity[18], so dose = 120-35 x wt (kg) x .714, e.g. 5 kg infant: 85 x 5 x .714 = 303 units)
PlaceboDrug
Also called Normal Saline
placebo (normal saline) after induction of anesthesia and before commencement of bypass. ATIII will be dosed using the formula recommended by the manufacturer: (goal activity - baseline activity) x weight (kg) x .714 (Assume: start with 35% activity [7], goal 120% activity[18], so dose = 120-35 x wt (kg) x .714, e.g. 5 kg infant: 85 x 5 x .714 = 303 units)
| Study group | Role | What they receive |
|---|---|---|
| Antithrombin III | Active comparator | Product- Antithrombin III is derived from pooled human plasma
ATIII will be dosed using the formula recommended by the manufacturer:
(goal activity - baseline activity) x weight (kg) x .714 (Assume: start with 35% activity [7], goal 120% activity[18], so dose = 120-35 x wt (kg) x .714, e.g. 5 kg infant: 85 x 5 x .714 = 303 units)
a. Receives: Drug: Placebo |
| Placebo | Placebo comparator | placebo (normal saline) Receives: Drug: Antithrombin III |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary hypothesis is that treatment with ATIII before bypass will decrease the activation of inflammation during CPB as measured by plasma neutrophil elastase.
Patients treated with ATIII as standard of care (good clinical practice) will remain in the study and be analyzed by their intention to treat group. Review of our clinical experience shows that treatment with ATIII for heparin resistance occurs in 2-3% of cases. If there are more than two patients so treated, we will also perform an "as-treated" analysis.
Measured: Expected average of 24 months
Secondary outcomes (6)
p-selectin
Measured: expected average of 24 months
interleukin-6
Measured: Expected average of 24 months
prothrombin fragment 1.2
Measured: expected average of 24 months
24 hour blood loss
Measured: 24 hours post surgery
ICU length of stay
Measured: Expected average of 3 days
Hospital length of stay
Measured: Expected average of 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2012 (estimated)
- Study startJul 2012
- Primary completionMar 2016
- Study completionMar 2016
Why it stopped: Challenges enrolling subjects in study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Heart DiseaseStudies of the same condition, open and under way.
- More trials run by University of RochesterThe 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Michael Eaton, University of Rochester
- Investigators
- Michael Eaton, MD, Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01698567
- Sponsor's protocol ID
- 38402
- Status last verified
- Mar 2016
- First submitted
- 4 Sep 2012
- Last updated
- 22 Mar 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Antithrombin III Supplementation for Infants Undergoing Cardiac Surgery With Cardiopulmonary Bypass.
- ClinicalTrials.gov ID
- NCT01698567
- Acronym / sponsor ID
- 38402
- Conditions
- Congenital Heart Disease
- Keywords
- Infants, Cardiopulmonary bypass, anticoaglution
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Challenges enrolling subjects in study
- Enrolment
- 6 participants (actual)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Study start
- Jul 2012
- Primary completion
- Mar 2016 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 3 Oct 2012 (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 discern whether supplementation of Antithrombin III will decrease coagulation and inflammation associated with cardiopulmonary bypass in infants undergoing cardiac surgery.
Detailed description
Cardiopulmonary bypass (CPB) and cardiovascular surgery initiate diffuse activation of coagulation, inflammation and fibrinolysis that often has deleterious effects on patient outcomes, including bleeding, transfusion, myocardial dysfunction, renal failure, pulmonary dysfunction, prolonged intubation, ICU and hospital length of stay, stroke and neurocognitive dysfunction, and mortality.Pediatric patients are especially at risk for hematologic derangement related to CPB. Not only are infants and children subject to the same diffuse activations of coagulation, inflammation, and fibrinolysis as adults, but their size, immaturity, and circulatory abnormalities secondary to congenital heart disease increase the risk of loss of hematologic homeostasis. Since an infant's blood volume is much smaller than that of the prime in the CPB pump, hemodilution alone produces impaired hemostasis related to thrombocytopenia and coagulation factor dilution The incidence of each of these complications is variable and depends on the diagnosis, operation, time on CPB and other factors.
Adequate anticoagulation during cardiopulmonary bypass (CPB) is essential to preserve the hemostatic system and ensure hemostasis after surgery. Heparin has long been the mainstay of anticoagulation for CPB, due to its ease of use, familiarity, and reversibility. For heparin to exert its anticoagulant effect, it must bind with an intrinsic cofactor, antithrombin III (ATIII) to inhibit enzymes of the intrinsic and final common coagulation pathways. It has been established that neonates have significantly decreased levels of ATIII relative to adults, and that this relative deficiency continues at least until 6 months of age. Children with congenital heart disease have further decreases in ATIII and other abnormalities of coagulation which may contribute to adverse outcomes. Given this ATIII deficiency, it is not surprising that heparin anticoagulation does not fully suppress coagulation during CPB. Neonates anticoagulated for CPB with heparin have ongoing activation of humoral and cellular coagulation with associated activation of inflammation and fibrinolysis. Improved anticoagulation may reduce activation of these cascades and improve outcomes. In addition, infants are at high risk for post cardiac surgery intervascular thrombosis. Baseline ATIII deficiency, and consumption of ATIII during bypass may contribute to a postoperative prothrombotic state esulting in this often fatal complication. ATIII supplementation may decrease this risk.
ATIII is available as a lyophilized product derived from pooled human plasma. Treatment with ATIII has been shown to improve the anticoagulant effects of heparin and attenuate activation of hemostasis and inflammation during adult CPB, and to decrease the incidence of thrombosis associated with central venous cannulation in children. The incidence of central venous thrombosis in infants undergoing cardiac surgery has been reported as 5.8 - 22% in neonates, with a resultant mortality of 20%.
The biologic half-life of antithrombin in healthy adult volunteers is 2.5 - 3.8 days. Pharmacokinetic data in neonates is not available, but biologic activity should certainly persist through the 2 highest risk periods:
CPB, where activation of coagulation produces activation of inflammation, both cellular and humoral, and fibrinolysis.
The early post-op period when patients typically become hypercoagulable as part of the stress response to surgery. Hypercoagulability places the patients at high risk for central line-associated thrombosis despite by heparin-containing flush solutions which are standard of care. Ensuring a near normal level of antithrombin appears to enhance the ability of the flush solutions to inhibit thrombin generation on the catheter.
ATIII has been used in infants after cardiac surgery to prophylax against central venous thrombosis, for infants with necrotizing enterocolitis (NEC), and as treatment for neonates with congenital ATIII deficiency. Neonates appear to respond as expected to ATIII supplementation, with clinical efficacy for central venous thrombosis but not NEC. No complications unique to infants were reported in any of these publications.
It is reasonable to expect that ATIII would be even more beneficial in infants less than 6 months old.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Antithrombin III | Active comparator | Drug: PlaceboProduct- Antithrombin III is derived from pooled human plasma ATIII will be dosed using the formula recommended by the manufacturer: (goal activity - baseline activity) x weight (kg) x .714 (Assume: start with 35% activity [7], goal 120% activity[18], so dose = 120-35 x wt (kg) x .714, e.g. 5 kg infant: 85 x 5 x .714 = 303 units) a. |
| Placebo | Placebo comparator | Drug: Antithrombin IIIplacebo (normal saline) |
Who may qualify
- Ages
- 1 day to 180 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Subjects will be included if they are presenting for elective cardiac surgery using cardiopulmonary bypass and are 180 days of age or less.
Key exclusion criteria (6 listed)
- Sensitivity to ATIII product (Thrombate, Grifols, Los Angeles, CA)
- Known inherited or acquired coagulation defect
- Current or prior treatment with pro-or anticoagulant medication within past 30 days (except aspirin or a single dose of heparin, e.g. for catheterization)
- Known central venous thrombosis
- Recent (30 days) transfusion with hemostatic blood products (fresh-frozen plasma, platelets, cryoprecipitate, whole blood)
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 |
|---|---|---|
| Antithrombin IIIAlso: ThrombateATIII will be dosed using the formula recommended by the manufacturer: (goal activity - baseline activity) x weight (kg) x .714 (Assume: start with 35% activity [7], goal 120% activity[18], so dose = 120-35 x wt (kg) x .714, e.g. 5 kg infant: 85 x 5 x .714 = 303 units) | Drug | Placebo |
| PlaceboAlso: Normal Salineplacebo (normal saline) after induction of anesthesia and before commencement of bypass. ATIII will be dosed using the formula recommended by the manufacturer: (goal activity - baseline activity) x weight (kg) x .714 (Assume: start with 35% activity [7], goal 120% activity[18], so dose = 120-35 x wt (kg) x .714, e.g. 5 kg infant: 85 x 5 x .714 = 303 units) | Drug | Antithrombin III |
Primary outcome
The primary hypothesis is that treatment with ATIII before bypass will decrease the activation of inflammation during CPB as measured by plasma neutrophil elastase.
Time frame: Expected average of 24 months
Patients treated with ATIII as standard of care (good clinical practice) will remain in the study and be analyzed by their intention to treat group. Review of our clinical experience shows that treatment with ATIII for heparin resistance occurs in 2-3% of cases. If there are more than two patients so treated, we will also perform an "as-treated" analysis.
6 secondary outcomes
p-selectin
Time frame: expected average of 24 months
interleukin-6
Time frame: Expected average of 24 months
prothrombin fragment 1.2
Time frame: expected average of 24 months
24 hour blood loss
Time frame: 24 hours post surgery
ICU length of stay
Time frame: Expected average of 3 days
Hospital length of stay
Time frame: Expected average of 3 weeks
Results and safety
The study was stopped early (reason given: Challenges enrolling subjects in study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2016.
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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.
Michael Eaton, MD principal investigator
University of Rochester
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 Antithrombin III in people with Congenital Heart Disease, run by University of Rochester 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 6 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: stopped early, as of the sponsor's last update on 22 Mar 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. Some participants in this study may receive a placebo. 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 stopped early (reason given: Challenges enrolling subjects in study). Results are not available in the source record.
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?
The primary hypothesis is that treatment with ATIII before bypass will decrease the activation of inflammation during CPB as measured by plasma neutrophil elastase. (time frame: Expected average of 24 months). 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 22 Mar 2016.