Trial Outcomes & Findings for Inhaled Tissue Plasminogen Activator for Acute Plastic Bronchitis (NCT NCT02315898)

NCT ID: NCT02315898

Last Updated: 2025-08-19

Results Overview

The number of subjects with new systemic and/or pulmonary and/or gross hematuria

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

Participants will be assessed every 24 hours (daily) for the duration of tPA treatment which was up to 4 days and at hospital discharge which typically occurred within 1 week of treatment initiation.

Results posted on

2025-08-19

Participant Flow

Participant milestones

Participant milestones
Measure
Treatment-inhaled tPA
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
PLE Controls
Participants with congenital heart disease and protein losing enteropathy but with no history of plastic bronchitis
Fontan Controls
Participants with Fontan physiology with no history of plastic bronchitis or PLE
Healthy Controls
Participants without congenital heart disease and no history of pulmonary disease (including plastic bronchitis) or protein losing enteropathy
Overall Study
STARTED
10
8
9
13
Overall Study
COMPLETED
8
8
9
12
Overall Study
NOT COMPLETED
2
0
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Treatment-inhaled tPA
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
PLE Controls
Participants with congenital heart disease and protein losing enteropathy but with no history of plastic bronchitis
Fontan Controls
Participants with Fontan physiology with no history of plastic bronchitis or PLE
Healthy Controls
Participants without congenital heart disease and no history of pulmonary disease (including plastic bronchitis) or protein losing enteropathy
Overall Study
2 in the treatment arm did not meet treatment eligibility; a healthy control screen failed
2
0
0
1

Baseline Characteristics

Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
PLE Controls
n=8 Participants
Participants with congenital heart disease and protein losing enteropathy but with no history of plastic bronchitis
Fontan Controls
n=9 Participants
Participants with Fontan physiology with no history of plastic bronchitis or PLE
Healthy Controls
n=12 Participants
Participants without congenital heart disease and no history of pulmonary disease (including plastic bronchitis) or protein losing enteropathy
Total
n=37 Participants
Total of all reporting groups
Age, Continuous
12.1 years
STANDARD_DEVIATION 4.1 • n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
13 years
STANDARD_DEVIATION 2.6 • n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
14 years
STANDARD_DEVIATION 3.2 • n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
11.7 years
STANDARD_DEVIATION 2.5 • n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
12.6 years
STANDARD_DEVIATION 3.3 • n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Sex: Female, Male
sex · Female
4 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
1 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
4 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
4 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
13 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Sex: Female, Male
sex · Male
4 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
7 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
5 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
8 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
24 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
2 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
2 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
6 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Ethnicity (NIH/OMB)
Not Hispanic or Latino
6 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
6 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
9 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
7 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
28 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
3 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
3 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
Asian
0 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
1 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
1 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
Black or African American
1 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
1 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
2 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
White
5 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
4 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
8 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
11 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
28 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
More than one race
2 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
3 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
5 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
0 Participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
1 Participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
1 Participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
Region of Enrollment
United States
8 participants
n=93 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
8 participants
n=4 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
9 participants
n=27 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
12 participants
n=483 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.
37 participants
n=36 Participants • Two participants in the Treatment arm were enrolled but were not treated because they did not have a subsequent episode of acute plastic bronchitis. One healthy control participant was enrolled and did not complete the study due to a screen failure.

PRIMARY outcome

Timeframe: Participants will be assessed every 24 hours (daily) for the duration of tPA treatment which was up to 4 days and at hospital discharge which typically occurred within 1 week of treatment initiation.

Population: The number of new, active systemic bleeding events were assessed for participants enrolled in the treatment arm only. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study.

The number of subjects with new systemic and/or pulmonary and/or gross hematuria

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Primary Endpoint: Number of Subjects That Develop New, Active Bleeding
0 Participants

SECONDARY outcome

Timeframe: Participants will be assessed at screening (if applicable), just prior to treatment and daily for the duration of tPA treatment, up to 4 days, at hospital discharge (~ 1 week) and again at 30 days.

Population: Oxygen saturation was assessed at screening (if applicable), prior to treatment and daily for the duration of tPA treatment, at hospital discharged and at 30 days. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study. Because pulse oximetry was measured at different times during hospitalization across patients, we report the mean pre-treatment and the hospital discharge (\~1 week post-treatment) mean (SD) values.

Changes in oxygen saturation (%) will be monitored by pulse oximetry (oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin in the blood). Since this measurement was made at different times for each participant, the mean (SD) oxygen saturation (%) prior to study drug administration (pre-treatment) and at hospital discharge (\~ 1 week post-treatment) was calculated.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Arterial Oxygen Saturation (%)
Pre-treatment
90.6 percent
Standard Deviation 4.7
Arterial Oxygen Saturation (%)
Post-treatment (hospital discharge)
90.6 percent
Standard Deviation 4.2

SECONDARY outcome

Timeframe: Participants will be assessed at screening (if applicable), just prior to treatment and then daily for the duration of tPA treatment, up to 4 days, at hospital discharge and again at 30 days.

Population: The FEV1 (L) was assessed prior to and during inhaled tPA treatment, at hospital discharge and at the 30 d follow up visit. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study. Many treatment arm participants did not have values at all time points.The outcome measure is the pre-treatment average, the overall average during treatment (days 0-4) and the average at hospital discharge (\~ 1 week) after treatment initiation.

The change in FEV1 (L) from pre- to post- tPA treatment will be assessed for each patient.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Forced Expiratory Volume in One Second (FEV1)
Pre-treatment
1.7 Liters
Standard Deviation 0.95
Forced Expiratory Volume in One Second (FEV1)
Treatment (days 0-4)
1.60 Liters
Standard Deviation 0.62
Forced Expiratory Volume in One Second (FEV1)
Hospital Discharge (~1 week)
1.42 Liters
Standard Deviation 0.66

SECONDARY outcome

Timeframe: Participants will be assessed at screening (if applicable), just prior to treatment and then daily for the duration of tPA treatment, up to 4 days, at hospital discharge and again at 30 days.

Population: The FEF25-75 will be assessed for each patient in the treatment arm. This is because the outcome measures were per-specified to collect data only from the treatment-inhaled tPA arm of the study. Many treatment arm participants did not have values at all time points.The outcome measure is the pre-treatment average, the overall average during treatment (days 0-4) and the average at hospital discharge (\~ 1 week) after treatment initiation.

The FEF25-75 will be assessed for each patient in the treatment arm prior to study drug, during study drug administration (days 0-4), at hospital discharge (\~ 1 week after treatment) and again at 30 days.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Forced Expiratory Flow 25-75% (FEF25-75)
Pre-treatment
1.23 Liters/second
Standard Deviation 0.2
Forced Expiratory Flow 25-75% (FEF25-75)
Treatment (days 0-4)
1.76 Liters/second
Standard Deviation 0.94
Forced Expiratory Flow 25-75% (FEF25-75)
Hospital discharge (~1 week)
1.14 Liters/second
Standard Deviation 0.09

SECONDARY outcome

Timeframe: Participants will be assessed at screening (if applicable), just prior to treatment and then daily for the duration of tPA treatment, up to 4 days, at hospital discharge and again at 30 days.

Population: The FVC (L) before, during and after tPA treatment will be assessed. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study. Many treatment arm participants did not have values at all time points.The outcome measure is the pre-treatment average, the overall average during treatment (days 0-4) and the average at hospital discharge (\~ 1 week) after treatment initiation.

The FVC (L) from prior to, during and after tPA treatment will be assessed for each patient.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Forced Vital Capacity (FVC)
Pre-treatment
2.0 Liters
Standard Deviation 1.14
Forced Vital Capacity (FVC)
Treatment (days 0-4)
1.91 Liters
Standard Deviation 0.66
Forced Vital Capacity (FVC)
Hospital discharge (~1 week)
1.80 Liters
Standard Deviation 0.65

SECONDARY outcome

Timeframe: Episodes of cast production will be assessed daily for the duration tPA treatment, up to 4 days and from hospital discharge (~1 week after treatment) up to 30 days

Population: Assessment of cast production during the study period was made in the PB treatment arm only. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study. This was based on self report. Cast sizes were not assessed or reported. The cumulative number of episodes of cast production reported at 30 days is reported.

Episodes of the production of airway casts by participants enrolled in the treatment arm will be assessed.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Frequency of Production/Expectoration of Airway Casts
9 episodes of cast production (30d)

SECONDARY outcome

Timeframe: A CXR will be acquired and assessed two times during the study- once just prior to the initiation of study drug and again at hospital discharge, up to ~1 week.

Population: All patients that received tPA. CXRs were scored by a pediatric pulmonologist using the Brasfield scoring system. A score of 25 represents a normal CXR. Points are deducted from 25 for abnormalities so the lower the score the greater the disease severity. This outcome was pre-specified for the treatment arm of the study only; CXRs were not obtained in the control arms.

tPA treatment-induced changes in the CXR will be assessed. The chest x-ray will be scored prior to and after tPA treatment at hospital discharge (\~1 week post treatment). The scores will be derived using the Brasfield scoring system which assesses air trapping, linear markings (bronchial wall thickness), bronchiectasis, lobar involvement and overall severity. A score of 25 represents a normal CXR. Points are deducted from 25 for abnormalities so the lower the score the greater the disease severity.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Changes in the Chest X-ray (CXR)
Pre-treatment
20.6 score on a scale (0-25 normal)
Standard Deviation 0.74
Changes in the Chest X-ray (CXR)
Post-treatment
20.9 score on a scale (0-25 normal)
Standard Deviation 0.90

SECONDARY outcome

Timeframe: Participants will be followed for the duration of tPA treatment, up to 4 days.

Population: The number of treatment arm participants who required urgent or emergent bronchoscopy was recorded. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study.

Requirement for urgent or emergent bronchoscopy during treatment was assessed.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Requirement for Urgent or Emergent Bronchoscopy
0 Participants

SECONDARY outcome

Timeframe: Participants will be followed for the duration of tPA treatment, up to 4 days.

Population: The number of participants in the treatment arm who required mechanical ventilation was recorded. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study.

Requirement for mechanical ventilation will be assessed in participants enrolled in the treatment arm during the treatment period.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Requirement for Mechanical Ventilation
0 Participants

SECONDARY outcome

Timeframe: Available (submitted) airway casts will assessed for the duration of the hospital stay, up to hospital discharge (~1 week).

Population: Any PB casts that were expectorated by treatment participants were sent for pathological analysis of casts by UM pathology. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study. None of the PB-tPA treated study participants produced and submitted airway casts for pathological assessment. Therefore, no data are available for this outcome measure.

PB cast fibrin and mucin content will be qualitatively assessed in casts that are collected before and after tPA treatment up to hospital discharge (\~1 week) by a co-investigator pathologist. Each cast will be qualitatively assessed for fibrin and mucin content based on standard pathological procedures.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: FDP will be assessed at screening (if applicable), prior to treatment and then daily during the hospital stay (~1 week) and again at 30 days

Population: Blood levels of FDP were assessed from participants in the treatment arm of the study. This is because the outcome measures were pre-specified to collect data only from the treatment-inhaled tPA arm of the study. The pre-treatment average of participants and the post-treatment average (at hospital discharge, \~ 1 week from the start of treatment) are reported.

Blood samples will be assayed for FDP (mg/L) during the study. This is a measure of fibrin degradation in the blood and is a value that can be altered by tPA treatment. An FDP (or D-dimer) value \<0.5 mg/L is considered normal.

Outcome measures

Outcome measures
Measure
Treatment-inhaled tPA
n=8 Participants
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
Detection of Fibrin Degradation Product (FDP) in the Systemic Circulation
Pre-treatment
0.32 mg/L
Standard Deviation 0.08
Detection of Fibrin Degradation Product (FDP) in the Systemic Circulation
Hospital discharge (~1 week)
4.14 mg/L
Standard Deviation 4.11

SECONDARY outcome

Timeframe: This measurement will be performed prior to tPA treatment, at hospital discharge (~ 1 week) and again at 30 days.

Population: The number of participants enrolled in the treatment arm who had a relevant change in CTQ-R questionnaire domain scores from pre-treatment to hospital discharge were not able to be assessed because there were no participants who completed questionnaires at these two time points. No data are available for this secondary outcome.

We will use a questionnaire to assess how many participants had changes in quality of life related to plastic bronchitis and its treatment during the study in participants enrolled in the treatment arm. For this, the Cystic Fibrosis Questionnaire-Revised (CFQ-R) will be used since there is not a specific plastic bronchitis questionnaire. The CFQ-R is designed to measure impact on overall health, daily life, perceived well-being and symptoms. The CFQ-R uses a Likert scale to rate nine quality of life domains: Physical, role/school, vitality, emotion, social, body image, eating, treatment burden, health perceptions and three symptom scales: Weight, respiratory, and digestion. Each item is summed to generate a domain score. Scores range from 0 to 100, with higher scores indicating better health.

Outcome measures

Outcome data not reported

Adverse Events

Treatment-inhaled tPA

Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths

PLE Controls

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Fontan Controls

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Healthy Controls

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Treatment-inhaled tPA
n=8 participants at risk
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
PLE Controls
n=8 participants at risk
Participants with congenital heart disease and protein losing enteropathy but with no history of plastic bronchitis
Fontan Controls
n=9 participants at risk
Participants with Fontan physiology with no history of plastic bronchitis or PLE
Healthy Controls
n=12 participants at risk
Participants without congenital heart disease and no history of pulmonary disease (including plastic bronchitis) or protein losing enteropathy
Congenital, familial and genetic disorders
serious adverse event
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.

Other adverse events

Other adverse events
Measure
Treatment-inhaled tPA
n=8 participants at risk
All patients with plastic bronchitis enrolled into the study will receive inhaled tPA. Treatment-inhaled tPA: Enrolled patients with acute plastic bronchitis (fibrin airway casts) will receive inhaled tPA treatment. The tPA regimen will consist of 5mg every six hours for a total of 72 hours.
PLE Controls
n=8 participants at risk
Participants with congenital heart disease and protein losing enteropathy but with no history of plastic bronchitis
Fontan Controls
n=9 participants at risk
Participants with Fontan physiology with no history of plastic bronchitis or PLE
Healthy Controls
n=12 participants at risk
Participants without congenital heart disease and no history of pulmonary disease (including plastic bronchitis) or protein losing enteropathy
Respiratory, thoracic and mediastinal disorders
hypoxemia
12.5%
1/8 • Number of events 2 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
nose bleed
25.0%
2/8 • Number of events 5 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
blood-tinged mucus
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
coughing
12.5%
1/8 • Number of events 4 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Cardiac disorders
Dizziness
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
emesis
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Vascular disorders
vasovagal response to venipuncture
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
8.3%
1/12 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Cardiac disorders
Atrial Tachycardia
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
Abdominal pain
25.0%
2/8 • Number of events 2 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
Ascites
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
Loose stools
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
Nausea
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
Poor appetite and constipation
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Gastrointestinal disorders
Vomiting
12.5%
1/8 • Number of events 2 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
General disorders
Febrile to 39.1
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
General disorders
Fever
25.0%
2/8 • Number of events 2 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
Erythematous and congested throat
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
Increased pleural effusion on chest x-ray
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
Nasal congestion
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
Patchy lobe atelectasis
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Respiratory, thoracic and mediastinal disorders
Rhinovirus
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Vascular disorders
Low blood pressure
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Blood and lymphatic system disorders
Abnormal labs on day 30 IgG, D-dimer
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Blood and lymphatic system disorders
Elevated D-dimer
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Blood and lymphatic system disorders
Hematuria
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Immune system disorders
Elevated WBC count
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
Immune system disorders
Systemic inflammatory response syndrome
12.5%
1/8 • Number of events 1 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/8 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/9 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.
0.00%
0/12 • 30 days
For the treatment arm of the study, AEs were systematic assessed during hospitalization and tPA treatment (measurements and routine clinical assessments for bleeding). Following hospital discharge, adverse events information collection was non-systematic, self-reporting. For the control participants, AEs were assessed at time of their one time study visit. These participants did not receive study drug.

Additional Information

Dr. Kathleen Stringer

University of Michigan

Phone: 734-647-4775

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place