Ignite Creation Date:
2024-05-06 @ 2:59 PM
Last Modification Date:
2024-10-26 @ 1:41 PM
Study NCT ID:
NCT04489251
Status:
UNKNOWN
Last Update Posted:
2021-11-22
First Post:
2020-07-21
Brief Title:
Assessment of the TGF-beta Pathway and Micro-RNA in Pediatric Pulmonary Arterial Hypertension
Sponsor:
Medical College of Wisconsin
Organization:
Medical College of Wisconsin
Study Overview
Official Title:
Assessment of the TGF-beta Pathway and Micro-RNA in Pediatric Pulmonary Arterial Hypertension
Status:
UNKNOWN
Status Verified Date:
2021-11
Last Known Status:
RECRUITING
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:
This is a prospective pilot study to assess the plasma levels of particular proteins involved in the transforming growth factor beta TGF-β pathway and its down stream regulators CHIP as well as micro RNA molecules in subjects with pulmonary arterial hypertension PAH and compare them to control subjects without PAH to see if they can be used as a diagnostic or prognostic marker of PAH and how this compares to other diagnostic biomarkers N-terminal pro-natriuretic peptide NT Pro-BNP and C-reactive protein CRP
Detailed Description:
Aim 1 This study will correlate proteins in the TGF- β signaling pathway and micro RNA levels with invasive catheterization and non-invasive echocardiography measurements of pulmonary artery pressures to assess for the presence and severity of PAH and compare these measurements to the established biomarkers of NT Pro BNP and CRP levels
Hypothesis 1 Plasma levels of proteins of the TGF-β pathway bone morphogenic protein BMP 2 4 6 7 9 and 10 along with activin A and TGF-β1 protein as well as CHIP carboxyl-terminus of Hsp70-intracting protein an enzyme that regulates the activations and exports of TGF- β to the nucleus will be significantly different in subjects with PH over control subjects
Hypothesis 2 Plasma levels of proteins in the TGF- β pathway BMP 2 4 6 7 9 and 10 along with activin A and TGF-β1 protein as well as CHIP will show better correlation with the presence of PAH and its severity than NT-Pro BNP and CRP levels
Hypothesis 3 The micro-RNA profiles in plasma will be significantly different in subjects with PAHPH over control subjects
Aim 2 To correlate proteinmicro-RNA levels with clinical status in PAH subjects as assessed by functional status exercise testing and PAH drug regimen to determine if they can correlate with disease severity
Hypothesis 1 Clinical findings in PAH patients will correlate with disease severity and study proteins and micro-RNA levels better than established biomarkers
Aim 3 To correlate evidence of genetic abnormalities through whole exome sequencing especially in regions known or suspected to cause PAH eg BMPR2 ENG and ALK1 mutations within the TGF-β pathway or lung development with the tested proteinmicro-RNA levels
Hypothesis 1 Genetic evaluation of patients with PAH will show abnormalities within the TGF-β pathway or lung development
Hypothesis 1 Plasma levels of proteins of the TGF-β pathway bone morphogenic protein BMP 2 4 6 7 9 and 10 along with activin A and TGF-β1 protein as well as CHIP carboxyl-terminus of Hsp70-intracting protein an enzyme that regulates the activations and exports of TGF- β to the nucleus will be significantly different in subjects with PH over control subjects
Hypothesis 2 Plasma levels of proteins in the TGF- β pathway BMP 2 4 6 7 9 and 10 along with activin A and TGF-β1 protein as well as CHIP will show better correlation with the presence of PAH and its severity than NT-Pro BNP and CRP levels
Hypothesis 3 The micro-RNA profiles in plasma will be significantly different in subjects with PAHPH over control subjects
Aim 2 To correlate proteinmicro-RNA levels with clinical status in PAH subjects as assessed by functional status exercise testing and PAH drug regimen to determine if they can correlate with disease severity
Hypothesis 1 Clinical findings in PAH patients will correlate with disease severity and study proteins and micro-RNA levels better than established biomarkers
Aim 3 To correlate evidence of genetic abnormalities through whole exome sequencing especially in regions known or suspected to cause PAH eg BMPR2 ENG and ALK1 mutations within the TGF-β pathway or lung development with the tested proteinmicro-RNA levels
Hypothesis 1 Genetic evaluation of patients with PAH will show abnormalities within the TGF-β pathway or lung development
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