129 Xenon MRI as a Biomarker for Diagnosis and Response to Therapy in Pulmonary Arterial Hypertension (PAH)
Overview
- Phase
- Phase 2
- Intervention
- 129Xe Hyperpolarized
- Conditions
- Not specified
- Sponsor
- Bastiaan Driehuys
- Enrollment
- 20
- Locations
- 2
- Primary Endpoint
- Pulmonary Vascular Remodeling
- Status
- Recruiting
- Last Updated
- 2 months ago
Overview
Brief Summary
The overall study objectives outlined in this study are to derive 129Xe MRI pulmonary vascular biomarker signatures that differentiate common subtypes of PAH and to determine the ability of 129Xe MRI to longitudinally monitor disease progression and response to therapy in PAH, with the aid of additional assessments, such as labs, echocardiography, and six-minute walk distance (6MWD).
Detailed Description
Subject Enrollment This study will consent and enroll 20 subjects total. • For Arm 1, 10 subjects with Idiopathic Pulmonary Arterial Hypertension (IPAH) will be consented and enrolled. For Arm 2, 10 subjects with Connective Tissue Disease Associated Pulmonary Arterial Hypertension (PAH-CTD) will be consented and enrolled. Study Design This study will be observational. Subjects in both arms of the trial will undergo a 129Xe MRI/MRS at timepoints of baseline, 3 months, 6 months, and 12 months. In addition to the this, data from standard of care assessments, such as labs, echocardiography, and six-minute walk distance (6MWD), will also collected at these timepoints. Primary Study Endpoints The primary endpoint for this trial will be the change in defect + low percentage of RBC signal on hyperpolarized 129Xe MRI from baseline to 12 months Secondary Study Endpoints There will be several secondary endpoints for this trial: * Change in regional and global RBC Oscillation Amplitudes on hyperpolarized 129Xe MR spectroscopy from baseline to 12 months * Change in 6MWD from baseline to 12 months * Change in NTproBNP from baseline to 12 months * Change in WHO FC from baseline to 12 months Primary Safety Endpoints There will be several primary safety endpoints for this trial: * Frequency of Adverse Events (AE) and/or Serious Adverse Events (SAE) * Withdrawals due to adverse event or death * Incidence of Adverse Events of Significant Interest (AESI): * Electrocardiogram and any findings * Physical examination and vital signs
Investigators
Bastiaan Driehuys
Professor of Radiology
Duke University
Eligibility Criteria
Inclusion Criteria
- •Arm 1 -IPAH
- •Age: 18-75 years
- •WHO functional class 2 or 3
- •Mean pulmonary artery pressures \> 20 mmHg
- •Pulmonary capillary wedge pressure ≤15 mmHg
- •Pulmonary vascular resistance \> 2 Wood Units (WU)
- •No other cause identified for PAH
- •Arm 2 -PAH-CTD
- •Age: 18-75 years
- •WHO functional class (FC) 2 or 3
Exclusion Criteria
- •PH other than Idiopathic PAH or PAH associated with CTD; any conditions that prevent the performance of 129Xe MRI scans will be excluded from the study.
Arms & Interventions
Idiopathic Pulmonary Arterial Hypertension
Arm 1... patients with IPAH
Intervention: 129Xe Hyperpolarized
Pulmonary Arterial Hypertension Associated with Connective Tissue Disease
Arm 2... patients with CTD-PAH
Intervention: 129Xe Hyperpolarized
Outcomes
Primary Outcomes
Pulmonary Vascular Remodeling
Time Frame: 1 year
The primary endpoint for this trial will be the change in defect + low percentage of RBC signal on hyperpolarized 129Xe MRI from baseline to 12 months
Secondary Outcomes
- RBC Oscillation Amplitude(1 year)
- 6 Minute Walk Distance(1 Year)
- NTproBNP(1 year)
- World Health Organization (WHO) Functional Class (FC)(1 year)