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

This study is currently recruiting participants.
Verified February 2013 by Penn State University
Sponsor:
Collaborator:
FDA Office of Orphan Products Development
Information provided by (Responsible Party):
Neal J. Thomas, Penn State University
ClinicalTrials.gov Identifier:
NCT00999713
First received: October 18, 2009
Last updated: February 8, 2013
Last verified: February 2013
History of Changes
  Purpose

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.


Condition Intervention Phase
Acute Lung Injury
 Drug: Calfactant
Other: Air placebo
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3 Trial of Calfactant for ALI in Pediatric Leukemia and HSCT Patients

Resource links provided by NLM:

MedlinePlus related topics: Leukemia
U.S. FDA Resources

Further study details as provided by Penn State University:

Primary Outcome Measures:
  • All-cause mortality at the time of PICU discharge [ Time Frame: PICU discharge ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Total duration of mechanical ventilation required during PICU admission. [ Time Frame: PICU discharge ] [ Designated as safety issue: No ]
  • Total duration of PICU and hospital stay required. [ Time Frame: Hospital discharge ] [ Designated as safety issue: No ]
  • Improvement in oxygenation, as measured by oxygenation index, in the initial 48 hours after treatment [ Time Frame: 48 hours after enrollment ] [ Designated as safety issue: No ]

Estimated Enrollment: 140
Study Start Date: June 2010
Estimated Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   18 Months to 21 Years
Genders Eligible for Study:   Both
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 mEq/L for more than 2 hours,
    • persistent arterial oxygen desaturation, PaO2 < 50 or SaO2saturation < 80%,
    • hyperkalemia, serum K+ > 6.5 plus widening of QRS complex on EKG
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00999713

Contacts
Contact: Neal J Thomas, MD, MSc 7175318521 nthomas@psu.edu
Contact: Robert F Tamburro, MD, MSc 7175318521 rtamburro@psu.edu

Locations
United States, Nebraska
The Nebraska Medical Center Recruiting
Omaha, Nebraska, United States
Contact: Edward Truemper            
Principal Investigator: Edward Truemper            
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States
Contact: Derek Wheeler, MD            
Principal Investigator: Derek Wheeler            
United States, Pennsylvania
Penn State College of Medicine, Penn State Milton S. Hershey Medical Center Recruiting
Hershey, Pennsylvania, United States, 17078
Contact: Neal J Thomas, MD, MSc     717-531-8521     nthomas@psu.edu    
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States
Contact: Laura Loftis, MD            
Principal Investigator: Laura Loftis, MD            
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States
Contact: Jennifer McArthur            
Principal Investigator: Jennifer McArthur            
Sponsors and Collaborators
Penn State University
FDA Office of Orphan Products Development
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
  More Information

Publications:

Responsible Party: Neal J. 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, R01 FD003410-01-A1
Study First Received: October 18, 2009
Last Updated: February 8, 2013
Health Authority: United States: Food and Drug Administration

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

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

ClinicalTrials.gov processed this record on May 23, 2013