Congenital Heart DiseasePhase 1NCT04181255
Cold Heart Study: A Randomized Pilot Trial of Surfactant Therapy
A Phase 1 study testing Curosurf in people with Congenital Heart Disease and Hypoplastic Left Heart Syndrome, run by University of Michigan at 1 site in United States.
Trial summary
This clinical trial is being done to see if giving surfactant (Curosurf®) will decrease the number of days that infants will need a breathing tube, decrease the days in the critical care unit and decrease the number of days needed in the hospital. Read more about this study
- Who is it for?
- Ages 1 day to 12 weeks
- Conditions the study is for
- Congenital Heart Disease, Hypoplastic Left Heart Syndrome
- What is being tested?
- Curosurf
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Feb 2020to 12 Jun 2020
- 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 12 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients less than or equal to 12 weeks of age with hypoplastic left heart syndrome (HLHS) undergoing cardiothoracic surgical correction (Norwood) requiring DHCA.
- Infants weighing 2500 grams or greater at the time of surgery.
- Written informed consent from parent(s) or legally appointed representative (LAR).
Who cannot join
- Patients who are less than 36 weeks Post Menstrual Age (PMA) at the time of surgical correction.
- Underlying craniofacial, airway or lung anomalies which could compromise administration of surfactant.
- Infants weighing less than 2500 grams at the time of the surgical correction.
- Mechanical ventilation for> 7 days prior to surgical correction.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Cold Heart Study: A Randomized Pilot Trial of Surfactant Therapy Following Deep Hypothermic Circulatory Arrest During Cardiac Surgery
This clinical trial is being done to see if giving surfactant (Curosurf®) will decrease the number of days that infants will need a breathing tube, decrease the days in the critical care unit and decrease the number of days needed in the hospital. The primary hypothesis for this study is that there will be fewer days needed on mechanical ventilation and improved lung compliance and pulmonary gas exchange.
Read the detailed description
This study will enroll infants undergoing cardiothoracic surgery requiring deep hypothermic circulatory arrest (DHCA) at the University of Michigan's C.S. Mott Children's Hospital. Patients that are eligible will be randomized to receive either one dose surfactant or the sham (air) arm after the surgical procedure. Patients will be monitored for an additional 30 days after the breathing tube is removed. It is possible that subjects may be discharged prior to the last assessment, therefore for subjects who have been discharged a member of the study team will contact the parent (s) or legal authorized representative by telephone.
- Being tested
- Curosurf, Sham
- Keywords
- Norwood procedure
- Medical subject headings
- Heart Defects, Congenital, Hypoplastic Left Heart Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CurosurfDrug
Also called poractant alfa, surfactant
This arm will consist of a single dose of Curosurf (200 mg/kg) that will be given in the Pediatric Cardiothoracic Unit (PCTU) after surgery once the subject has reached a normalized body temperature.
ShamDrug
This arm will consist of a single dose of sham (air) that will be given through the endotracheal tube in the Pediatric Cardiothoracic Intensive Care Unit after surgery once the subject has reached a normalized body temperature.
| Study group | Role | What they receive |
|---|---|---|
| Curosurf | Experimental | Not described Receives: Drug: Curosurf |
| Sham (air) | Placebo comparator | Not described Receives: Drug: Sham |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of days of mechanical ventilation
This will be the number of days from intubation to the extubation date.
Measured: Throughout hospitalization (approximate average 5 days)
Number of days in the Pediatric Cardiothoracic Unit
From time of admission until transfer out of the unit.
Measured: Approximately 11 days
Number of post-operative hospital days
Measured: Up to 1 year
Secondary outcomes (9)
Total number of patients requiring thoracostomy tube placement for air leak not associated with standard post-operative management prior to extubation
Post-surgery prior to extubation.
Measured: Approximately 1 week
Total number of patients requiring high frequency ventilation (HFV) (High frequency oscillator ventilation or High frequency jet ventilation)
Post-surgery throughout hospitalization.
Measured: Up to 1 year
Total number of patients requiring extracorporeal membrane oxygenation (ECMO) for pulmonary failure
Post-surgery throughout hospitalization.
Measured: Up to 1 year
Changes in positive end-expiratory pressures post intervention
After therapy until extubation.
Measured: Baseline to approximately 1 week
Changes in peak inspiratory pressures post intervention
After therapy until extubation.
Measured: Baseline to approximately 1 week
Changes in dynamic lung compliance post intervention
After therapy until extubation.
Measured: Baseline to approximately 1 week
Changes in oxygen requirements post intervention
After therapy until extubation.
Measured: Baseline to approximately 1 week
Changes in oxygenation index (OI) post intervention
OI is the product of mean airway pressure and fraction of inspired oxygen divided by the partial pressure of oxygen. After therapy until extubation.
Measured: Baseline to approximately 1 week
Time to successful extubation readiness trials (ERT) post intervention
Measured in hours until successful ERT, and then successful extubation. Not everyone who passes an ERT is immediately extubated (typically done once per day), so the plan is daily.
Successful extubation is defined as remaining extubated for 48 hours.
Measured: Approximately 1 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2019
- Study start25 Feb 2020
- Primary completion12 Jun 2020
- Study completion12 Jun 2020
Why it stopped: Investigator Retirement
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 MichiganThe 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
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Michigan
- Sponsor type
- University, hospital or other
- Collaborators
- Chiesi Farmaceutici S.p.A.
- Responsible party
- Principal investigator, Steven Donn, M.D., University of Michigan
- Investigators
- Steven Donn, MD, Principal investigator, University of Michigan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04181255
- Sponsor's protocol ID
- HUM00160492
- Status last verified
- Sep 2023
- First submitted
- 26 Nov 2019
- Last updated
- 13 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Cold Heart Study: A Randomized Pilot Trial of Surfactant Therapy Following Deep Hypothermic Circulatory Arrest During Cardiac Surgery
- ClinicalTrials.gov ID
- NCT04181255
- Acronym / sponsor ID
- HUM00160492
- Keywords
- Norwood procedure
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Investigator Retirement
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of MichiganUniversity, hospital or other
- Collaborators
- Chiesi Farmaceutici S.p.A.
- Study start
- 25 Feb 2020 (actual)
- Primary completion
- 12 Jun 2020 (actual)
- Study completion
- 12 Jun 2020 (actual)
- First posted
- 29 Nov 2019 (actual)
- 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.
This clinical trial is being done to see if giving surfactant (Curosurf®) will decrease the number of days that infants will need a breathing tube, decrease the days in the critical care unit and decrease the number of days needed in the hospital. The primary hypothesis for this study is that there will be fewer days needed on mechanical ventilation and improved lung compliance and pulmonary gas exchange.
Detailed description
This study will enroll infants undergoing cardiothoracic surgery requiring deep hypothermic circulatory arrest (DHCA) at the University of Michigan's C.S. Mott Children's Hospital. Patients that are eligible will be randomized to receive either one dose surfactant or the sham (air) arm after the surgical procedure. Patients will be monitored for an additional 30 days after the breathing tube is removed. It is possible that subjects may be discharged prior to the last assessment, therefore for subjects who have been discharged a member of the study team will contact the parent (s) or legal authorized representative by telephone.
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 |
|---|---|---|
| Curosurf | Experimental | Drug: Curosurf |
| Sham (air) | Placebo comparator | Drug: Sham |
Who may qualify
- Ages
- 1 day to 12 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Patients less than or equal to 12 weeks of age with hypoplastic left heart syndrome (HLHS) undergoing cardiothoracic surgical correction (Norwood) requiring DHCA.
- Infants weighing 2500 grams or greater at the time of surgery.
- Written informed consent from parent(s) or legally appointed representative (LAR).
Key exclusion criteria (4 listed)
- Patients who are less than 36 weeks Post Menstrual Age (PMA) at the time of surgical correction.
- Underlying craniofacial, airway or lung anomalies which could compromise administration of surfactant.
- Infants weighing less than 2500 grams at the time of the surgical correction.
- Mechanical ventilation for> 7 days prior to surgical correction.
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 |
|---|---|---|
| CurosurfAlso: poractant alfa, surfactantThis arm will consist of a single dose of Curosurf (200 mg/kg) that will be given in the Pediatric Cardiothoracic Unit (PCTU) after surgery once the subject has reached a normalized body temperature. | Drug | Curosurf |
| ShamThis arm will consist of a single dose of sham (air) that will be given through the endotracheal tube in the Pediatric Cardiothoracic Intensive Care Unit after surgery once the subject has reached a normalized body temperature. | Drug | Sham (air) |
Primary outcome
Number of days of mechanical ventilation
Time frame: Throughout hospitalization (approximate average 5 days)
This will be the number of days from intubation to the extubation date.
Number of days in the Pediatric Cardiothoracic Unit
Time frame: Approximately 11 days
From time of admission until transfer out of the unit.
Number of post-operative hospital days
Time frame: Up to 1 year
9 secondary outcomes
Total number of patients requiring thoracostomy tube placement for air leak not associated with standard post-operative management prior to extubation
Time frame: Approximately 1 week
Total number of patients requiring high frequency ventilation (HFV) (High frequency oscillator ventilation or High frequency jet ventilation)
Time frame: Up to 1 year
Total number of patients requiring extracorporeal membrane oxygenation (ECMO) for pulmonary failure
Time frame: Up to 1 year
Changes in positive end-expiratory pressures post intervention
Time frame: Baseline to approximately 1 week
Changes in peak inspiratory pressures post intervention
Time frame: Baseline to approximately 1 week
Changes in dynamic lung compliance post intervention
Time frame: Baseline to approximately 1 week
Changes in oxygen requirements post intervention
Time frame: Baseline to approximately 1 week
Changes in oxygenation index (OI) post intervention
Time frame: Baseline to approximately 1 week
Time to successful extubation readiness trials (ERT) post intervention
Time frame: Approximately 1 week
Results and safety
The study was stopped early (reason given: Investigator Retirement). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2023.
- Individual participant dataSharing planned: No
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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.
Steven Donn, MD principal investigator
University of Michigan
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 Curosurf in people with Congenital Heart Disease and Hypoplastic Left Heart Syndrome, run by University of Michigan 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 4 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 13 Sep 2023. 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: Investigator Retirement). 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?
Number of days of mechanical ventilation (time frame: Throughout hospitalization (approximate average 5 days)). The study lists 3 primary outcomes in all. 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 13 Sep 2023.