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

NCT ID: NCT00999713

Last Updated: 2018-03-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 43 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-06-30
• Study Completion Date: 2015-10-31

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)

Conditions

Acute Lung Injury

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Calfactant

Endotracheal calfactant administration

Group Type: EXPERIMENTAL

Calfactant

Intervention Type: DRUG

Endotracheal calfactant, up to 3 doses if subject qualifies

Placebo (air)

Endotracheal air administration

Group Type: PLACEBO_COMPARATOR

Air placebo

Intervention Type: OTHER

Endotracheal air administration

Interventions

Calfactant

Endotracheal calfactant, up to 3 doses if subject qualifies

Intervention Type: DRUG

Air placebo

Endotracheal air administration

Intervention Type: OTHER

Eligibility 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).

• Minimum Eligible Age: 18 Months
• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Milton S. Hershey Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Neal Thomas

Neal J. Thomas, MD, Penn State College of Medicine, Penn State Milton S. Hershey Medical Center

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Neal J Thomas, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Penn State College of Medicine, Penn State Milton S. Hershey Medical Center

Robert F Tamburro, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Penn State College of Medicine, Penn State Milton S. Hershey Medical Center

Locations

Phoenix Children's Hospital

Phoenix, Arizona, United States

Children's Hospital of Los Angeles

Los Angeles, California, United States

University of California San Francisco

San Francisco, California, United States

Riley Children's Hospital

Indianapolis, Indiana, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Weill Cornell Medical Center

New York, New York, United States

Maria Fareri Children's Hospital

Valhalla, New York, United States

Rainbow Babies Hospital

Cleveland, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Penn State College of Medicine, Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Texas Children's Hospital

Houston, Texas, United States

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Hospital Sainte Justine

Montreal, Quebec, Canada

Countries

United States; Canada

References

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.

Reference Type: BACKGROUND

PMID: 18679142 (View on PubMed)

Willson 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.

Reference Type: BACKGROUND

PMID: 15671432 (View on PubMed)

Other Identifiers

R01FD003410-01A1

Identifier Type: FDA

Identifier Source: secondary_id

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1R01FD003410-01

Identifier Type: FDA

Identifier Source: org_study_id

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