NCT00999713

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)

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
43

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jun 2010

Longer than P75 for phase_2

Geographic Reach
2 countries

16 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 18, 2009

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 22, 2009

Completed
7 months until next milestone

Study Start

First participant enrolled

June 1, 2010

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2015

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2015

Completed
2.5 years until next milestone

Results Posted

Study results publicly available

March 16, 2018

Completed
Last Updated

March 16, 2018

Status Verified

February 1, 2018

Enrollment Period

5.3 years

First QC Date

October 18, 2009

Results QC Date

January 19, 2018

Last Update Submit

February 15, 2018

Conditions

Acute Lung Injury

Keywords

Acute Lung InjuryCancerPediatrics

Outcome Measures

Primary Outcomes (1)

  • All-cause Mortality at the Time of Pediatric Intensive Care Unit (PICU) Discharge

    Overall mortality rate from admission to PICU discharge

    Admission to PICU discharge, up to 120 days

Secondary Outcomes (4)

  • Ventilator Free Days (VFDs)

    60 days after study enrollment

  • Total Duration of Stay Required

    Admission to discharge, up to 120 days

  • Change in Oxygenation: First Intervention

    48 hours after enrollment, up to 12 hours after each intervention

  • Change in Oxygenation: Second Intervention

    48 hours after enrollment, up to 12 hours after each intervention

Study Arms (2)

Calfactant

EXPERIMENTAL

Endotracheal calfactant administration

Drug: Calfactant

Placebo (air)

PLACEBO COMPARATOR

Endotracheal air administration

Other: Air placebo

Interventions

CalfactantDRUG

Endotracheal calfactant, up to 3 doses if subject qualifies

Calfactant
Air placeboOTHER

Endotracheal air administration

Placebo (air)

Eligibility Criteria

Age18 Months - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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),
  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, United States

Location

University of California San Francisco

San Francisco, California, United States

Location

Riley Children's Hospital

Indianapolis, Indiana, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, United States

Location

Weill Cornell Medical Center

New York, New York, United States

Location

Maria Fareri Children's Hospital

Valhalla, New York, United States

Location

Rainbow Babies Hospital

Cleveland, Ohio, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, United States

Location

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

Hershey, Pennsylvania, 17078, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Location

Texas Children's Hospital

Houston, Texas, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Location

Hospital Sainte Justine

Montreal, Quebec, Canada

Location

Related Publications (2)

  • 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.

    PMID: 18679142BACKGROUND

  • 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.

    PMID: 15671432BACKGROUND

MeSH Terms

Conditions

Acute Lung InjuryNeoplasms

Interventions

calfactant

Condition Hierarchy (Ancestors)

Lung InjuryLung DiseasesRespiratory Tract Diseases

Results Point of Contact

Title
Neal J. Thomas, M.D.
Organization
Penn State College of Medicine
nthomas@psu.edu
717-531-5337

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Neal J. Thomas, MD, Penn State College of Medicine, Penn State Milton S. Hershey Medical Center

Study Record Dates

First Submitted

October 18, 2009

First Posted

October 22, 2009

Study Start

June 1, 2010

Primary Completion

September 1, 2015

Study Completion

October 1, 2015

Last Updated

March 16, 2018

Results First Posted

March 16, 2018

Record last verified: 2018-02

Locations

US(15)CA(1)