Ignite Creation Date:
2024-07-17 @ 10:44 AM
Last Modification Date:
2024-10-26 @ 3:33 PM
Study NCT ID:
NCT06475976
Status:
RECRUITING
Last Update Posted:
2024-06-26
First Post:
2024-05-23
Brief Title:
Multidimensional Phenotype Classification in Grade 3 Bronchopulmonary Dysplasia
Sponsor:
Childrens Hospital of Philadelphia
Organization:
Childrens Hospital of Philadelphia
Study Overview
Official Title:
Multidimensional Phenotype Classification in Grade 3 Bronchopulmonary Dysplasia
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Bronchopulmonary Dysplasia BPD or chronic lung disease of prematurity is the most consequential complication of preterm birth and is strong predictor of childhood pulmonary and neurodevelopmental disability particularly in infants diagnosed with grade 3 BPD ventilator dependence at 36 weeks postmenstrual age the most severe disease form This study aims to 1 generate the first empirically defined phenotype classification system for grade 3 BPD developed using a rich array of objective and quantitative cardiopulmonary diagnostic clinical and biological data and 2 define the association between phenotype subgroups and neurodevelopmental and respiratory outcomes through 2 years corrected age
Detailed Description:
Bronchopulmonary Dysplasia BPD or infant chronic lung disease is the most consequential morbidity of prematurity It affects 50 of extremely preterm infants 30wk gestation and can incur 1 million in costs per child Among infants who develop grade 3 BPD most severe grade defined as invasive ventilation at 36 weeks postmenstrual age nearly 80 suffer life-long respiratory impairment and 60 suffer severe developmental disability Rates of grade 3 BPD are increasing and no proven therapies treat this disease A key contributor to these gaps is the nearly singular reliance on the prescribed respiratory support to define BPD severity select therapies and assess prognosis This subjective diagnostic approach masks heterogeneity in clinical presentation treatment responsiveness and outcomes In other heterogenous lung diseases such as chronic obstructive pulmonary disease cystic fibrosis and asthma evidence-based phenotyping identification of patient subgroups based on shared characteristics objectively classifies disease sub-types improves patient counseling promotes discovery of novel pathological mechanisms and leads to more effective phenotype-targeted therapies The central hypothesis of the present study is that deep multidimensional phenotyping in grade 3 BPD is feasible with existing diagnostic technologies will reliably characterize disease heterogeneity and will improve outcome prediction Confirmation of this hypothesis holds promise to promote a frameshift towards objective diagnostic approaches and first-of-their-kind phenotype-specific trials in infants with BPD
Existing preliminary data support the feasibility of phenotyping in grade 3 BPD and suggest newer diagnostic techniques may improve disease characterization Using data from lung computed tomography scan cardiac echo and bronchoscopy researchers showed that preterm infants with grade 3 BPD can be classified into phenotypes based on the presence or absence of severe parenchymal lung disease abnormal large airways and pulmonary arterial hypertension This classification scheme correlated with pre-discharge outcomes and suggested possible phenotype-specific therapies Recent discoveries indicate that serial quantitative cardiopulmonary imaging and evaluation of mechanistic contributors to BPD including lung inflammation gastroesophageal reflux recurrent hypoxemia and lung microbial dysbiosis may improve disease phenotyping and prediction of childhood neurodevelopmental and respiratory outcomes This study builds on this information and uses multidimensional imaging biological and clinical data plus robust statistical techniques to propose an objective phenotype classification system for grade 3 BPD
Enrolled infants will undergo baseline quantitative chest computed tomography with angiography CTA cardiac echocardiography bronchoscopy with lavage 24-hour esophageal pH-impedance testing pulmonary mechanics testing oximetry and complete medical record review at enrollment Repeat diagnostic testing will be performed 6-8wk later and cardiopulmonary monitoring and outcome data collected until discharge These data will be used to empirically define phenotypes and assess phenotype stability Enrolled participants will undergo validated neurodevelopmental and respiratory assessments through 2 years corrected age The diagnostic performance the empirically defined phenotype classification system for predicting 2 year outcomes will be determined
Existing preliminary data support the feasibility of phenotyping in grade 3 BPD and suggest newer diagnostic techniques may improve disease characterization Using data from lung computed tomography scan cardiac echo and bronchoscopy researchers showed that preterm infants with grade 3 BPD can be classified into phenotypes based on the presence or absence of severe parenchymal lung disease abnormal large airways and pulmonary arterial hypertension This classification scheme correlated with pre-discharge outcomes and suggested possible phenotype-specific therapies Recent discoveries indicate that serial quantitative cardiopulmonary imaging and evaluation of mechanistic contributors to BPD including lung inflammation gastroesophageal reflux recurrent hypoxemia and lung microbial dysbiosis may improve disease phenotyping and prediction of childhood neurodevelopmental and respiratory outcomes This study builds on this information and uses multidimensional imaging biological and clinical data plus robust statistical techniques to propose an objective phenotype classification system for grade 3 BPD
Enrolled infants will undergo baseline quantitative chest computed tomography with angiography CTA cardiac echocardiography bronchoscopy with lavage 24-hour esophageal pH-impedance testing pulmonary mechanics testing oximetry and complete medical record review at enrollment Repeat diagnostic testing will be performed 6-8wk later and cardiopulmonary monitoring and outcome data collected until discharge These data will be used to empirically define phenotypes and assess phenotype stability Enrolled participants will undergo validated neurodevelopmental and respiratory assessments through 2 years corrected age The diagnostic performance the empirically defined phenotype classification system for predicting 2 year outcomes will be determined
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R01HL168066 | NIH | None | None |