A Study for the Identification of Biomarker Signatures for Diagnosis of Pulmonary Hypertension (PH) in Participants at Low or Intermediate Probability of PH Undergoing Cardiac Magnetic Resonance Imaging

NCT04480723 | Completed Early Phase 1

• 2 other identifiers: CR108827NAPUH0003
• Study Type: interventional
• Target: 86
• Locations: 2 countries
• Sites: 8

Brief Summary

The primary purpose of this study is to estimate the percentage of participants in the population considered clinically as not having pulmonary hypertension (PH) that are positive for PH biomarker (micro ribonucleic acid \[RNA\]); estimate the percentage of participants in the population considered clinically as not having PH that are positive for PH by cardiac magnetic resonance imaging (MRI) and compare results of the MRI to the biomarker results; to determine the performance of the biomarker signatures identified in the CIPHER (NAPUH0001) study in terms of sensitivity, specificity, positive predictive value (PPV) and negative predictive value (NPV), in a population considered clinically as not having PH.

Conditions

Hypertension, Pulmonary

Outcome Measures

Primary Outcomes (4)

• Percentage of Participants in the Population Considered Clinically as not Having PH that are Positive for PH Biomarker

  Percentage of (transthoracic echocardiography \[TTE\] low/intermediate probability) participants who are positive for the biomarker will be estimated.

  Up to 90 Days

• Percentage of Participants Considered Clinically as not Having PH that are Positive for PH by Cardiac MRI

  Percentage of participants considered clinically as not having PH that are positive for PH by cardiac magnetic resonance imaging (MRI) will be evaluated.

  Up to 90 Days

• Performance of Biomarker Results

  The performance of the biomarker in the diagnosis of PH will be evaluated using the MRI result.

  Up to 90 Days

• Performance of the Biomarker Signatures Identified in the CIPHER Study in Terms of Sensitivity, specificity, positive predictive value (PPV) and Negative Predictive Value (NPV), in Participants not Having PH

  Performance of the biomarker signatures identified in the CIPHER study in terms of sensitivity, specificity, PPV and NPV, in a population considered clinically as not having PH will be estimated.

  Up to 90 Days

Secondary Outcomes (1)

• Biomarker Data Pooled with the Biomarker Data Collected from the CIPHER (NAPUH0001) Study

  Up to 90 Days

Study Arms (1)

Participants with Low or Intermediate Probability of PH

OTHER

Participants who underwent a work-up for the suspicion of PH that includes transthoracic echocardiography (TTE) and who were considered to have a low or intermediate probability of PH according to TTE (local interpretation) will be enrolled. Blood samples will be taken and a cardiac magnetic resonance imaging (MRI) will be performed to evaluate the presence of Pulmonary Hypertension (PH). The TTEs that were performed by local standards will be collected and undergo central interpretation using European society of Cardiology / European respiratory society (ESC/ERS) guidelines to confirm the local interpretation.

Other: Blood Sample; Diagnostic Test: Cardiac MRI

Interventions

Blood Sample OTHER

Blood samples will be taken and analyzed to evaluate the presence of PH.

Participants with Low or Intermediate Probability of PH

Cardiac MRI DIAGNOSTIC_TEST

Cardiac MRI will be performed to evaluate the presence of PH.

Participants with Low or Intermediate Probability of PH

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Referred for diagnostic work-up for pulmonary hypertension (PH)
• With low or intermediate (but not high) probability of PH by transthoracic echocardiography (TTE) according to European Society of Cardiology (ESC)/ European Respiratory Society (ERS) Guidelines if at the time of enrollment Right Heart Catheterization (RHC) is not deemed to be clinically indicated
• Medically stable on the basis of physical examination, medical history and vital signs performed at screening. Any abnormalities must be consistent with the underlying illness in the study population and this determination must be recorded in the participant's source documents and initialed by the investigator
• Must sign an Informed consent (ICF) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study
• Must sign a separate approval in the ICF if he or she agrees to provide an optional (DNA) sample for research. Refusal to give consent for the optional (DNA) research sample does not exclude a participant from participation in the study

You may not qualify if:

• Undergone RHC within 2 years
• Participants requiring renal dialysis
• Participants post-lung or heart transplant
• Severe left ventricular dysfunction: left ventricular ejection fraction less then (\<) 35 percent
• Ongoing Contagious respiratory disease

Sponsors & Collaborators

• Actelion: lead

Study Sites (8)

Universitatsklinikum Bonn

Bonn, 53105, Germany

Location

Universitaetsklinikum Giessen

Giessen, 35392, Germany

Location

Universitatsklinikum Schleswig Holstein

Lübeck, 23538, Germany

Location

Royal United Hospital

Bath, BA1 3NG, United Kingdom

Location

National Waiting Times Centre Board Golden Jubilee National Hospital

Glasgow, G81 4DY, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Hammersmith Hospital

London, W12 0HS, United Kingdom

Location

Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital

Sheffield, S10 2RX, United Kingdom

Location

MeSH Terms

Conditions

Hypertension, Pulmonary

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Actelion Clinical Trial

  Actelion

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2020
• First Posted: July 21, 2020
• Study Start: December 11, 2020
• Primary Completion: May 13, 2022
• Study Completion: May 18, 2022
• Last Updated: March 30, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share

Locations

DE (3); GB (5)