Acute Lung InjuryPhase 2/3CALIPSONCT00999713
CALIPSO: Calfactant for Acute Lung Injury in Pediatric Stem Cell Transplant and Oncology Patients
A Phase 2/3 study testing Calfactant in people with Acute Lung Injury, run by Milton S. Hershey Medical Center at 16 sites in 2 countries.
Trial summary
Acute lung injury (ALI) is a common, life-threatening complication among pediatric leukemia and lymphoma and hematopoietic stem cell transplant (HSCT) recipients. Although these children represent a relatively small and unique patient population, they account for the largest proportion of deaths of all pediatric… Read more about this study
- Who is it for?
- Ages 18 months to 21 years
- Conditions the study is for
- Acute Lung Injury
- What is being tested?
- Calfactant
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 16 sitesin 2 countries
- Study dates
- Jun 2010to Oct 2015
- Phase
- Phase 2/3
- 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 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must meet criteria for acute lung injury
- Intubated, mechanically ventilated, with respiratory failure secondary to diffuse, bilateral parenchymal lung disease (as judged by chest x-ray).
- Oxygenation index (OI) > 13, but < 37, for two consecutive blood gases which should be separated by at least one hour within 48 hours of the initiation of mechanical ventilation.
- Arterial catheter placement
- Parental informed consent
- Patients must have a diagnosis of leukemia/lymphoma undergoing active treatment or following HSCT for any indication. Leukemia/lymphoma will be defined according to the National Cancer Institute Surveillance Epidemiology and End Results Collaborative Staging Manual including those conditions defined as borderline such as myelodysplastic syndromes. All forms of HSCT will be eligible, allogeneic as well as autologous.
Who cannot join
- Clinical diagnosis of congestive heart failure and/or pulmonary capillary wedge pressure >15 mmHg, or uncorrected congenital heart disease.
- Glasgow Coma Score < 8 (prior to respiratory failure).
- Pre-existing limitations on care options, (Do Not Attempt Resuscitation Orders, etc).
- Patients with impending death from another disease.
- Patients moribund or with other organ failure at possible randomization:
- hypotension unresponsive to treatment (mean BP < 60 or < 5th % for age),
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- persistent cardiac tachyarrhythmia >150/minute, or persistent bradyarrythmia < 50/minute, or age appropriate criteria for younger children,
- metabolic acidosis > - 10 milliequivalent (mEq)/L for more than 2 hours,
- persistent arterial oxygen desaturation, arterial partial pressure of oxygen (PaO2) < 50 or oxygen saturation (SaO2) saturation < 80%,
- hyperkalemia, serum K+ > 6.5 plus widening of QRS complex on EKG (QRS complex corresponds to the depolarization of the right and left ventricles of the heart).
Trial locations and contacts
16 sites in 2 countries.
16 sites in 2 countries
United States15 sites
Rainbow Babies Hospital
Cleveland, Ohio
Nationwide Children's Hospital
Columbus, Ohio
Hackensack University Medical Center
Hackensack, New Jersey
Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17078
Texas Children's Hospital
Houston, Texas
Riley Children's Hospital
Indianapolis, Indiana
Children's Hospital of Los Angeles
Los Angeles, California
St. Jude Children's Research Hospital
Memphis, Tennessee
Children's Hospital of Wisconsin
Milwaukee, Wisconsin
Weill Cornell Medical Center
New York, New York
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
Phoenix Children's Hospital
Phoenix, Arizona
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania
University of California San Francisco
San Francisco, California
Maria Fareri Children's Hospital
Valhalla, New York
About this study
Official title: A Phase 3 Trial of Calfactant for ALI in Pediatric Leukemia and HSCT Patients
Acute lung injury (ALI) is a common, life-threatening complication among pediatric leukemia and lymphoma and hematopoietic stem cell transplant (HSCT) recipients. Although these children represent a relatively small and unique patient population, they account for the largest proportion of deaths of all pediatric diseases. The long-term goal of this project is to improve outcomes among these patients. Recently, the intratracheal administration of calfactant has resulted in decreased mortality among children with ALI including promising results among children with cancer and following HSCT. Consequently, the primary specific aim of this study is to assess the effect of calfactant on intensive care (PICU) survival among pediatric leukemia and lymphoma and HSCT patients with ALI. Secondary aims include assessment of the effect of calfactant on oxygenation and on the length of mechanical ventilation, PICU stay, and hospital stay. Calfactant therapy has been found to be of benefit in acute lung injury in the overall pediatric population by improving oxygenation and decreasing mortality. These findings, in conjunction with recent subgroup analysis in which calfactant therapy appeared to improve outcomes in immunocompromised children provide the rationale for assessing calfactant therapy in this patient population.
Funding Source - FDA Office of Orphan Products Development (OOPD)
- Conditions
- Acute Lung Injury
- Being tested
- Calfactant, Air placebo
- Keywords
- Acute Lung Injury, Cancer, Pediatrics
- Medical subject headings
- Acute Lung Injury, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CalfactantDrug
Endotracheal calfactant, up to 3 doses if subject qualifies
Air placeboOther
Endotracheal air administration
| Study group | Role | What they receive |
|---|---|---|
| Calfactant | Experimental | Endotracheal calfactant administration Receives: Drug: Calfactant |
| Placebo (air) | Placebo comparator | Endotracheal air administration Receives: Other: Air placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
All-cause Mortality at the Time of Pediatric Intensive Care Unit (PICU) Discharge
Overall mortality rate from admission to PICU discharge
Measured: Admission to PICU discharge, up to 120 days
Secondary outcomes (4)
Ventilator Free Days (VFDs)
Number of days the patient is alive and off of the ventilator
Measured: 60 days after study enrollment
Total Duration of Stay Required
Length of stay (LOS) ,measured in days, from admission to PICU discharge and admission to hospital discharge.
Measured: Admission to discharge, up to 120 days
Change in Oxygenation: First Intervention
The Oxygenation Index after the first intervention is calculated as the fraction of inspired oxygen, in percent, times the mean airway pressure, in mmHg, divided by the partial pressure of oxygen in arterial blood, in mmHg. Lower values are better.
Measured: 48 hours after enrollment, up to 12 hours after each intervention
Change in Oxygenation: Second Intervention
The Oxygenation Index after the second intervention (if applicable) is calculated as the fraction of inspired oxygen, in percent, times the mean airway pressure, in mmHg, divided by the partial pressure of oxygen in arterial blood, in mmHg. Lower values are better.
Measured: 48 hours after enrollment, up to 12 hours after each intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2009 (estimated)
- Study startJun 2010
- Primary completionSep 2015
- Study completionOct 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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Acute Lung InjuryStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey Medical CenterThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Neal Thomas, Penn State University
- Investigators
- Neal J Thomas, MD, MSc, Principal investigator, Penn State College of Medicine, Penn State Milton S. Hershey Medical CenterRobert F Tamburro, MD, MSc, Principal investigator, Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00999713
- Sponsor's protocol ID
- 1R01FD003410-01
- Other IDs
- R01FD003410-01A1
- Acronym
- CALIPSO
- Status last verified
- Feb 2018
- First submitted
- 18 Oct 2009
- Last updated
- 16 Mar 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Tamburro RF, Thomas NJ, Pon S, Jacobs BR, Dicarlo JV, Markovitz BP, Jefferson LS, Willson DF; Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network. Post hoc analysis of calfactant use in immunocompromised children with acute lung injury: Impact and feasibility of further clinical trials. Pediatr Crit Care Med. 2008 Sep;9(5):459-64. doi: 10.1097/PCC.0b013e3181849bec.
PubMed 18679142Willson DF, Thomas NJ, Markovitz BP, Bauman LA, DiCarlo JV, Pon S, Jacobs BR, Jefferson LS, Conaway MR, Egan EA; Pediatric Acute Lung Injury and Sepsis Investigators. Effect of exogenous surfactant (calfactant) in pediatric acute lung injury: a randomized controlled trial. JAMA. 2005 Jan 26;293(4):470-6. doi: 10.1001/jama.293.4.470.
PubMed 15671432
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Trial of Calfactant for ALI in Pediatric Leukemia and HSCT Patients
- ClinicalTrials.gov ID
- NCT00999713
- Acronym / sponsor ID
- CALIPSO / 1R01FD003410-01
- Conditions
- Acute Lung Injury
- Keywords
- Acute Lung Injury, Cancer, Pediatrics
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- Jun 2010
- Primary completion
- Sep 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 22 Oct 2009 (estimated)
- Results
- Posted 16 Mar 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Acute lung injury (ALI) is a common, life-threatening complication among pediatric leukemia and lymphoma and hematopoietic stem cell transplant (HSCT) recipients. Although these children represent a relatively small and unique patient population, they account for the largest proportion of deaths of all pediatric diseases. The long-term goal of this project is to improve outcomes among these patients. Recently, the intratracheal administration of calfactant has resulted in decreased mortality among children with ALI including promising results among children with cancer and following HSCT. Consequently, the primary specific aim of this study is to assess the effect of calfactant on intensive care (PICU) survival among pediatric leukemia and lymphoma and HSCT patients with ALI. Secondary aims include assessment of the effect of calfactant on oxygenation and on the length of mechanical ventilation, PICU stay, and hospital stay. Calfactant therapy has been found to be of benefit in acute lung injury in the overall pediatric population by improving oxygenation and decreasing mortality. These findings, in conjunction with recent subgroup analysis in which calfactant therapy appeared to improve outcomes in immunocompromised children provide the rationale for assessing calfactant therapy in this patient population.
Funding Source - FDA Office of Orphan Products Development (OOPD)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Calfactant | Experimental | Drug: CalfactantEndotracheal calfactant administration |
| Placebo (air) | Placebo comparator | Other: Air placeboEndotracheal air administration |
Who may qualify
- Ages
- 18 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Patients must meet criteria for acute lung injury
- Patients must have a diagnosis of leukemia/lymphoma undergoing active treatment or following HSCT for any indication. Leukemia/lymphoma will be defined according to the National Cancer Institute Surveillance Epidemiology and End Results Collaborative Staging Manual including those conditions defined as borderline such as myelodysplastic syndromes. All forms of HSCT will be eligible, allogeneic as well as autologous.
Key exclusion criteria (10 listed)
- Clinical diagnosis of congestive heart failure and/or pulmonary capillary wedge pressure >15 mmHg, or uncorrected congenital heart disease.
- Glasgow Coma Score < 8 (prior to respiratory failure).
- Pre-existing limitations on care options, (Do Not Attempt Resuscitation Orders, etc).
- Patients with impending death from another disease.
- Patients moribund or with other organ failure at possible randomization:
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 |
|---|---|---|
| CalfactantEndotracheal calfactant, up to 3 doses if subject qualifies | Drug | Calfactant |
| Air placeboEndotracheal air administration | Other | Placebo (air) |
Primary outcome
All-cause Mortality at the Time of Pediatric Intensive Care Unit (PICU) Discharge
Time frame: Admission to PICU discharge, up to 120 days
Overall mortality rate from admission to PICU discharge
4 secondary outcomes
Ventilator Free Days (VFDs)
Time frame: 60 days after study enrollment
Total Duration of Stay Required
Time frame: Admission to discharge, up to 120 days
Change in Oxygenation: First Intervention
Time frame: 48 hours after enrollment, up to 12 hours after each intervention
Change in Oxygenation: Second Intervention
Time frame: 48 hours after enrollment, up to 12 hours after each intervention
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 18679142Tamburro RF, Thomas NJ, Pon S, Jacobs BR, Dicarlo JV, Markovitz BP, Jefferson LS, Willson DF; Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network. Post hoc analysis of calfactant use in immunocompromised children with acute lung injury: Impact and feasibility of further clinical trials. Pediatr Crit Care Med. 2008 Sep;9(5):459-64. doi: 10.1097/PCC.0b013e3181849bec.
- Background: PubMed 15671432Willson DF, Thomas NJ, Markovitz BP, Bauman LA, DiCarlo JV, Pon S, Jacobs BR, Jefferson LS, Conaway MR, Egan EA; Pediatric Acute Lung Injury and Sepsis Investigators. Effect of exogenous surfactant (calfactant) in pediatric acute lung injury: a randomized controlled trial. JAMA. 2005 Jan 26;293(4):470-6. doi: 10.1001/jama.293.4.470.
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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.
Neal J Thomas, MD, MSc principal investigator
Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
Robert F Tamburro, MD, MSc principal investigator
Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Calfactant in people with Acute Lung Injury, run by Milton S. Hershey Medical Center at 16 sites in 2 countries.
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 6 inclusion and 10 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 16 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Yes. Results are posted in the registry (first posted 16 Mar 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?
All-cause Mortality at the Time of Pediatric Intensive Care Unit (PICU) Discharge (time frame: Admission to PICU discharge, up to 120 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00999713, which also lists 2 linked publications.
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 16 Mar 2018.