ClinicalTrials.gov
ClinicalTrials.gov Menu

CALIPSO: Calfactant for Acute Lung Injury in Pediatric Stem Cell Transplant and Oncology Patients (CALIPSO)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00999713
Recruitment Status : Completed
First Posted : October 22, 2009
Results First Posted : March 16, 2018
Last Update Posted : March 16, 2018
Sponsor:
Information provided by (Responsible Party):
Neal Thomas, Penn State University

Brief 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 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)


Condition or disease Intervention/treatment Phase
Acute Lung Injury Drug: Calfactant Other: Air placebo Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 43 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3 Trial of Calfactant for ALI in Pediatric Leukemia and HSCT Patients
Study Start Date : June 2010
Actual Primary Completion Date : September 2015
Actual Study Completion Date : October 2015


Arm Intervention/treatment
Experimental: Calfactant
Endotracheal calfactant administration
Drug: Calfactant
Endotracheal calfactant, up to 3 doses if subject qualifies

Placebo Comparator: Placebo (air)
Endotracheal air administration
Other: Air placebo
Endotracheal air administration




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Ventilator Free Days (VFDs) [ Time Frame: 60 days after study enrollment ]
    Number of days the patient is alive and off of the ventilator

  2. Total Duration of Stay Required [ Time Frame: Admission to discharge, up to 120 days ]
    Length of stay (LOS) ,measured in days, from admission to PICU discharge and admission to hospital discharge.

  3. Change in Oxygenation: First Intervention [ Time Frame: 48 hours after enrollment, up to 12 hours after each 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.

  4. Change in Oxygenation: Second Intervention [ Time Frame: 48 hours after enrollment, up to 12 hours after each 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Months to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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
  2. 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.

Exclusion Criteria:

  1. Clinical diagnosis of congestive heart failure and/or pulmonary capillary wedge pressure >15 mmHg, or uncorrected congenital heart disease.
  2. Glasgow Coma Score < 8 (prior to respiratory failure).
  3. Pre-existing limitations on care options, (Do Not Attempt Resuscitation Orders, etc).
  4. Patients with impending death from another disease.
  5. Patients moribund or with other organ failure at possible randomization:

    • hypotension unresponsive to treatment (mean BP < 60 or < 5th % for age),
    • 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00999713


Locations
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States
United States, California
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California San Francisco
San Francisco, California, United States
United States, Indiana
Riley Children's Hospital
Indianapolis, Indiana, United States
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States
United States, New York
Weill Cornell Medical Center
New York, New York, United States
Maria Fareri Children's Hospital
Valhalla, New York, United States
United States, Ohio
Rainbow Babies Hospital
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
United States, Pennsylvania
Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17078
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
United States, Texas
Texas Children's Hospital
Houston, Texas, United States
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Canada, Quebec
Hospital Sainte Justine
Montreal, Quebec, Canada
Sponsors and Collaborators
Milton S. Hershey Medical Center
Investigators
Principal Investigator: Neal J Thomas, MD, MSc Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
Principal Investigator: Robert F Tamburro, MD, MSc Penn State College of Medicine, Penn State Milton S. Hershey Medical Center

Publications:
Responsible Party: Neal Thomas, Neal J. Thomas, MD, Penn State College of Medicine, Penn State Milton S. Hershey Medical Center, Penn State University
ClinicalTrials.gov Identifier: NCT00999713     History of Changes
Other Study ID Numbers: 1R01FD003410-01 ( U.S. FDA Grant/Contract )
R01FD003410-01A1 ( U.S. FDA Grant/Contract )
First Posted: October 22, 2009    Key Record Dates
Results First Posted: March 16, 2018
Last Update Posted: March 16, 2018
Last Verified: February 2018

Keywords provided by Neal Thomas, Penn State University:
Acute Lung Injury
Cancer
Pediatrics

Additional relevant MeSH terms:
Wounds and Injuries
Lung Injury
Acute Lung Injury
Respiratory Distress Syndrome, Adult
Lung Diseases
Respiratory Tract Diseases
Thoracic Injuries
Respiration Disorders
Calfactant
Pulmonary Surfactants
Respiratory System Agents