A Study for the Identification of Biomarker Signatures for Early Detection of Pulmonary Hypertension (PH)

NCT04193046 | Completed Early Phase 1

• 2 other identifiers: CR108723NAPUH0001
• Study Type: interventional
• Target: 907
• Locations: 9 countries
• Sites: 49

Brief Summary

The primary purpose of this study is to identify and develop biomarker signatures based on circulating micro ribonucleic acid (RNA) in the blood samples associated with high risk of pulmonary hypertension (PH) to assist in the diagnosis of PH; to estimate the sensitivity, specificity, positive predictive value, and negative predictive value of the biomarker signatures in identifying participants with PH by comparing the biomarker signatures to right heart catheterization (RHC) and to compare the sensitivity, specificity, positive predictive value, and negative predictive value of the biomarker signatures with the sensitivity, specificity, positive predictive value, and negative predictive value of transthoracic echocardiogram (TTE) in identifying participants with PH documented by RHC.

Conditions

Hypertension, Pulmonary

Outcome Measures

Primary Outcomes (4)

• Expression Levels of miRNAs Biomarkers

  Blood samples will be collected for micro Ribonucleic acid (miRNA) assay by qPCR to identify a biomarker signature for the early detection of pulmonary hypertension (PH).

  Day 1

• Biomarker signatures for miRNA

  Blood will be collected for miRNA to identify a biomarker signature(s) for detection of participants at high risk of PH.

  Day 1

• Biomarker Signature Performance in Identifying Participants with PH

  Biomarker signature performance will be evaluated by estimating sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) of the biomarker signatures documented by RHC for identifying participants with PH.

  Day 1

• Comparison of Performance of Biomarker Signatures and Transthoracic Echocardiogram (TTE) Based on Sensitivity, Specificity, Positive Predictive Value, and Negative Predictive Value

  The performance of the biomarkers signatures will be compared to the performance of TTE to identify participants with PH documented by RHC via sensitivity, specificity, positive predictive value, and negative predictive value. The sensitivity/specificity of the tests will be compared using McNemar's test.

  Day 1

Secondary Outcomes (2)

• Area Under ROC Curve in Participants with PH who are Receiving Therapies Versus not Receiving drug Therapy

  Day 1

• Area Under ROC Curve in Participants with PAH, CTEPH, Isolated pre-Capillary and post-Capillary PH

  Day 1

Study Arms (1)

Participants with PH and non-PH

OTHER

Blood samples will be collected for biomarker analysis from new (incident) and existing (prevalent) participants who undergo right heart catheterization (RHC). Participants will be categorized into non-PH or PH based on the results of the RHC and those who are found to have PH will be further classified into the different groups of PH. A transthoracic echocardiography (TTE) will be performed if not done previously.

Other: Blood Sample

Interventions

Blood Sample OTHER

Prevalent (existing) and incident (new) participants who are found to either have PH or not by RHC will provide blood sample for the establishment of a biomarker signature of PH.

Participants with PH and non-PH

Eligibility Criteria

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

You may qualify if:

• Having undergone an right heart catheterization (RHC) within 18 months (prevalent PH participants) or 6 months (prevalent non-PH participants) or have undergone or planned RHC within 6 weeks (incident participants). The results of the incident RHC (incident participants) or the most recent RHC (prevalent participants) will be used to classify the participant in one of the above study population categories
• 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 provide an Informed Consent Form (ICF) (or their legally acceptable representative must sign) indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study
• Must provide a separate informed consent (or their legally-acceptable representative must sign) if he or she agrees to provide an optional (deoxyribonucleic acid \[DNA\]) sample for research (where local regulations permit). 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:

• Participants requiring renal dialysis
• History of lung or heart transplant (waiting list status or consideration of enlisting is allowed)
• Severe left ventricular dysfunction: Left ventricular ejection function less then (\<) 35 percent (%)

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (49)

University of Southern California

Los Angeles, California, 90033, United States

Location

University Of Colorado Cardiac And Vascular Center

Aurora, Colorado, 80045-2548, United States

Location

Ascension St. Vincent's Lung Institute

Jacksonville, Florida, 32204, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

University Of Iowa - Hospitals & Clinics

Iowa City, Iowa, 52242, United States

Location

Norton Pulmonary Specialists

Louisville, Kentucky, 40202, United States

Location

LSU Health Sciences Center New Orleans

New Orleans, Louisiana, 70112, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111-1552, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198-5990, United States

Location

Pulmonary Health Physicians, PC

Liverpool, New York, 13088, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390-8550, United States

Location

The Houston Methodist Research Institute

Houston, Texas, 77030, United States

Location

Pulmonary Associates Of Richmond

Richmond, Virginia, 23230, United States

Location

ULB Erasme Ziekenhuis

Brussels, 1070, Belgium

Location

UZ Leuven Gasthuisberg

Leuven, 3000, Belgium

Location

CHU de Brest - Hopital de la Cavale Blanche

Brest, 29200, France

Location

Hopital de Bicetre

Le Kremlin-Bicêtre, 94270, France

Location

Hôpital Cardiologique - Chru Lille

Lille, 59037, France

Location

CHU de Montpellier - Arnaud de Villeneuve

Montpellier, 34295, France

Location

Hopital Larrey CHU de Toulouse

Toulouse, 31059, France

Location

Universitatsklinikum Bonn

Bonn, 53105, Germany

Location

Universitätsklinikum Carl Gustav Carus Medizinische Klinik und Poliklinik 1-Pneumologie

Dresden, 01307, Germany

Location

Universitaetsklinikum Giessen

Giessen, 35392, Germany

Location

Thoraxklinik Heidelberg

Heidelberg, 69126, Germany

Location

Lungenfachklinik Immenhausen

Immenhaus, 34376, Germany

Location

Universitätsklinikum Jena Klinik für Innere Medizin I

Jena, 07747, Germany

Location

Universitatsklinikum Schleswig Holstein

Lübeck, 23538, Germany

Location

VUMC Amsterdam

Amsterdam, 1007 MB, Netherlands

Location

Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego

Lublin, 20 718, Poland

Location

Europejskie Centrum Zdrowia Otwock Sp z o o

Otwock, 05 400, Poland

Location

Samodzielny Publiczny Szpital Kliniczny nr 2 PUM

Szczecin, 70-111, Poland

Location

Hosp Univ Vall D Hebron

Barcelona, 8035, Spain

Location

Hosp Clinic de Barcelona

Barcelona, 8036, Spain

Location

Hosp. Univ. 12 de Octubre

Madrid, 28041, Spain

Location

Hosp Clinico Univ de Salamanca

Salamanca, 37008, Spain

Location

Hosp. Univ. Marques de Valdecilla

Santander, 39008, Spain

Location

Hosp. Virgen de La Salud

Toledo, 45004, Spain

Location

CNE 'Cherkasy Regional Cardiological Center of Cherkasy Regional Council'

Cherkasy, 18009, Ukraine

Location

CE 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiosurgery'

Dnipro, 49070, Ukraine

Location

SI National Scientific Center Institute of Cardiology of M.D. Strazhesko of NAMS of Ukraine

Kyiv, 02000, Ukraine

Location

SI 'National Institute of Phtisiology and Pulmonology n.a. F.G. Yanovsky of NAMS of Ukraine'

Kyiv, 03038, Ukraine

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

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  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: December 6, 2019
• First Posted: December 10, 2019
• Study Start: December 16, 2019
• Primary Completion: December 20, 2021
• Study Completion: February 18, 2022
• Last Updated: September 12, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (5); UA (4); BE (2); DE (7); NL (1); GB (5); PL (3); ES (6); US (16)