Trial Outcomes & Findings for 'TOTAL' (Tracheal Occlusion To Accelerate Lung Growth) Trial (NCT NCT02875860)
NCT ID: NCT02875860
Last Updated: 2022-06-23
Results Overview
The null hypothesis to be tested is that there is no difference in survival between fetuses managed expectantly during pregnancy versus those undergoing antenatal therapy (FETO).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
At hospital discharge, an average of 1.5 months
Results posted on
2022-06-23
Participant Flow
Participant milestones
| Measure |
Standardized Postnatal Care (Expectant)
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
3
|
|
Overall Study
COMPLETED
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Standardized Postnatal Care (Expectant)
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
'TOTAL' (Tracheal Occlusion To Accelerate Lung Growth) Trial
Baseline characteristics by cohort
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=3 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
32.5 years
STANDARD_DEVIATION 2.1 • n=5 Participants
|
32.3 years
STANDARD_DEVIATION 7.6 • n=7 Participants
|
32.4 years
STANDARD_DEVIATION 5.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
BMI
|
21.5 kg/m²
STANDARD_DEVIATION 0.7 • n=5 Participants
|
27.6 kg/m²
STANDARD_DEVIATION 1.8 • n=7 Participants
|
25.2 kg/m²
STANDARD_DEVIATION 3.6 • n=5 Participants
|
|
Observed to Expected Lung to Head Ratio at Enrollment
|
35.5 percentage
n=5 Participants
|
31 percentage
n=7 Participants
|
31 percentage
n=5 Participants
|
|
Gestational Age at Delivery
|
39.2 weeks
n=5 Participants
|
39.0 weeks
n=7 Participants
|
39.0 weeks
n=5 Participants
|
|
Liver Herniation present at Enrollment
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At hospital discharge, an average of 1.5 monthsPopulation: Intent to Treat Population (all participants assigned to Expectant management or FETO).
The null hypothesis to be tested is that there is no difference in survival between fetuses managed expectantly during pregnancy versus those undergoing antenatal therapy (FETO).
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=3 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Proportion of Neonate Survival at Discharge From Hospital
|
1 Participants
|
3 Participants
|
PRIMARY outcome
Timeframe: At 6 months of agePopulation: Intent to Treat Population (all participants assigned to Expectant management or FETO) of all subjects who survived to discharge from the hospital (primarly outcome #1).
The number of survivors requiring supplemental oxygen at 6 months of age
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=3 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Participants Requiring Supplemental Oxygen
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: at 6 months of agePopulation: As-treated ('per protocol') analysis. 1 patient in the expectant management arm died and is excluded from this analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Measured as FiO2 (oxygen) amount required as a grade (0-III) with Grade 0 indicating the best outcome and Grade III indicating the worst outcome. Grade 0 = No Bronchopulmonary Dysplasia (BPD); Grade I = FiO2 21% or room air; Grade II = FiO2 22-29%; Grade III = FiO2 \>29%, CPAP or mechanical ventilation.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Grade of Oxygen Dependency
0 = No Bronchopulmonary Dysplasia (BPD)
|
0 Participants
|
1 Participants
|
|
Grade of Oxygen Dependency
II = FiO2 22-29%
|
1 Participants
|
1 Participants
|
|
Grade of Oxygen Dependency
III = FiO2 >29%, CPAP or mechanical ventilation
|
0 Participants
|
0 Participants
|
|
Grade of Oxygen Dependency
I = FiO2 21% or room air
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: During the first 4 weeks of life (neonatal period).Population: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Occurrence of severe pulmonary hypertension in the neonatal period.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Occurrence of Severe Pulmonary Hypertension
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Neonatal period (during the first 4 weeks of life)Population: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Need for extracorporeal membrane oxygenation (ECMO) in the neonatal period
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
ECMO (Extracorporeal Membrane Oxygenation) Support
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Measured in neonate at delivery by MRI and/or ultrasoundPopulation: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Postnatal grade classification (A-D) using CDH study group standardized system with A being the smallest defects and D being the largest defects. A = Defect entirely surrounded by muscle; B = Small (\<50%) portion of the chest wall devoid of diaphragm tissue; C = Large (\>50%) portion of the chest wall devoid of diaphragm tissue; D = Complete or near complete absence of the diaphragm.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
CDH Defect Size
A: Entirely surrounded by muscle
|
0 Participants
|
0 Participants
|
|
CDH Defect Size
C: Large (>50%) portion of the chest wall devoid of diaphragm tissue
|
2 Participants
|
1 Participants
|
|
CDH Defect Size
D: Complete or near complete absence of the diaphragm
|
0 Participants
|
0 Participants
|
|
CDH Defect Size
B: Small (<50%) portion of the chest wall devoid of diaphragm tissue
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: At the time of discharge from the NICU, an average of 1.5 monthsPopulation: As-treated ('per protocol') analysis. 1 patient in the expectant management arm died and is excluded from this analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Length of stay in the neonatal intensive care unit measured in days
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Number of Days in the NICU
|
39 days
|
53 days
Interval 49.0 to 57.0
|
SECONDARY outcome
Timeframe: During the first 4 weeks of life (neonatal period)Population: As-treated ('per protocol') analysis. 1 patient in the expectant management arm died and is excluded from this analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Length of time participants required ventilator support measured in days.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Ventilatory Support
|
18 days
|
21 days
Interval 15.0 to 27.0
|
SECONDARY outcome
Timeframe: During first 2 months of lifePopulation: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
As measured by presence in medical record ≤ 2 months postnatally by ultrasound (yes/no)
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Number of Subjects With Periventricular Leukomalacia (PVL)
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: During the first 4 weeks of life (neonatal period)Population: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
As measured by presence in medical record
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Neonatal Sepsis
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: During first month of lifePopulation: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Measured as presence in neonate during first month by MRI and/or ultrasound.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Intraventricular Hemorrhage
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: At the time of discharge from the NICU, an average of 1.5 monthsPopulation: As-treated ('per protocol') analysis. 1 patient in the expectant management arm died and is excluded from this analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Postnatal grade classification presence of grade III or higher using standardized system (yes/no)
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Retinopathy of Prematurity
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: At hospital discharge, an average of 1.5 monthsPopulation: As-treated ('per protocol') analysis. 1 patient in the expectant management arm died and is excluded from this analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
The number of days until full enteral feeding
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Days to Full Enteral Feeding
|
27 days
|
29.5 days
Interval 23.0 to 36.0
|
SECONDARY outcome
Timeframe: At the time of discharge from the NICU, an average of 1.5 monthsPopulation: As-treated ('per protocol') analysis. 1 patient in the expectant management arm died and is excluded from this analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
Presence of reflux above 1/3 of esophagus on clinically indicated radiologic exam
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=1 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Gastroesophageal Reflux
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: From the time of birth until discharge from the NICUPopulation: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
How many days from birth until the surgery is performed to repair the defect.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
CDH (Congenital Diaphragmatic Hernia) Surgery Repair
|
1 days
Interval 1.0 to 1.0
|
2.5 days
Interval 2.0 to 3.0
|
SECONDARY outcome
Timeframe: At the time of the surgical repair postnatally, up to 3 days postnatalPopulation: As-treated ('per protocol') analysis. 1 patient from the FETO arm withdrew prior to receiving the FETO procedure and is excluded from this analysis.
The number of participants who had a patch used in the repair of the CDH defect.
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=2 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Use of Patch in CDH Repair
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: At 24 months of agePopulation: Intent to Treat Population (all participants assigned to Expectant management or FETO).
The number of participants that survived to 24 months of age
Outcome measures
| Measure |
Standardized Postnatal Care (Expectant)
n=2 Participants
Mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
Prenatal Intervention (FETO)
n=3 Participants
Patients will undergo fetoscopic endoluminal tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in the expectant . In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days.
GoldBal2 detachable balloon: Placement of the balloon using the plug/unplug method.
Baltaccidbpe100 Delivery Catheter: The catheter assists with implanting the balloon in the plug/unplug method.
Standardized postnatal care: After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
|
|---|---|---|
|
Survival
|
1 Participants
|
3 Participants
|
Adverse Events
Standardized Postnatal Care (Expectant)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 1 deaths
Prenatal Intervention (FETO)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place