Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT04955990 | PAH | United States
Previous Study | Return to List | Next Study

A Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants (CARE PAH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04955990
Recruitment Status : Recruiting
First Posted : July 9, 2021
Last Update Posted : July 20, 2022
Sponsor:
Information provided by (Responsible Party):
Actelion

Brief Summary:
The purpose of this study is to describe the time to all-cause death and time to death due to pulmonary arterial hypertension (PAH) or first hospitalization due to PAH in the overall study population and within each cohort.

Condition or disease Intervention/treatment Phase
Pulmonary Arterial Hypertension Drug: PAH Therapies Phase 4

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3000 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: An International, Non-Drug Interventional, Real-world Cohort of PAH Patients Newly Initiating PAH Therapy With Guideline-directed Assessments of Disease Severity
Actual Study Start Date : October 14, 2021
Estimated Primary Completion Date : March 15, 2027
Estimated Study Completion Date : March 29, 2030


Arm Intervention/treatment
Experimental: Participants with PAH
Participants with pulmonary arterial hypertension (PAH) who newly initiate any PAH therapy(ies) at the index date (date when a participant starts the first new PAH therapy after baseline assessments) in a routine clinical setting, either as first-line therapy, as replacement therapies, as concomitant with other PAH therapies, or have already been receiving macitentan 10 milligrams (mg) for at least 3 months prior to the index date. The primary data source for this study will be the medical records of each participant.
Drug: PAH Therapies
PAH therapies will be administered in a routine clinical setting and data will be collected from participants who newly initiate any PAH therapy(ies), or have already been receiving macitentan 10 mg for at least 3 months prior to the index date. Participants will receive PAH therapies such as Macitentan, Ambrisentan, Bosentan, Tadalafil, Sildenafil, Riociguat, Selexipag, Epoprostenol, Iloprost, Treprostinil, and Beraprost.




Primary Outcome Measures :
  1. Time to all Cause Death [ Time Frame: Up to 6 years ]
    Time to all cause death will be reported. All-cause death defined as deaths due to any cause.

  2. Time to Death due to Pulmonary Arterial Hypertension (PAH) or First Hospitalization due to PAH [ Time Frame: Up to 6 years ]
    Time to death due to PAH or first hospitalization due to PAH will be reported.


Secondary Outcome Measures :
  1. Time to Death due to PAH [ Time Frame: Up to 6 years ]
    Time to death due to PAH will be reported.

  2. Time to First all-cause Hospitalization [ Time Frame: Up to 6 years ]
    Time to first all-cause hospitalization will be reported.

  3. Time to First Morbidity/Mortality Event [ Time Frame: Up to 6 years ]
    Time to first morbidity/mortality event will be reported. All-cause death, non-planned hospitalization due to PAH (including for worsening of PAH, atrial septostomy, lung transplantation with or without heart transplantation, or initiation of parenteral prostacyclins), PAH-related disease progression, defined as (both criteria must be satisfied): At least 15 percent (%) decrease in 6-minute walking distance (6MWD) from baseline or therapy change visit, and initiation of additional PAH therapy or worsening of World Health Organization (WHO) functional class (FC) will be collected for morbidity/mortality events assessment.

  4. Time to Clinical Worsening [ Time Frame: Up to 6 years ]
    Time to clinical worsening will be reported. All-cause death, non-planned hospitalization due to PAH (including for worsening of PAH, atrial septostomy, lung transplantation with or without heart transplantation, or initiation of parenteral prostacyclins), PAH-related deterioration identified by at least 1 criterion: worsening of WHO FC, deterioration by at least 15% in exercise capacity, as measured by the 6MWD, any signs or symptoms of right-sided heart failure will be collected for clinical worsening assessment.

  5. Medical Resource Utilization [ Time Frame: Up to 6 years ]
    Number per year of all-cause and PAH-related hospitalizations; number per year of in-patient hospital days for all causes and PAH-related causes; number per year of emergency room visits for all causes and for PAH-related causes that do not result in hospital admittance will be collected for assessment of medical resource utilization.

  6. Change from Baseline in 6-minute Walk Distance (6MWD) [ Time Frame: Baseline up to 6 years ]
    Change from baseline in 6MWD according to non-invasive criteria will be assessed as per the low (greater than [>] 440 meters [m]), intermediate (165-440m), and high-risk category (less than [<] 165m).

  7. Change from Baseline in World Health Organization (WHO) Functional Class (FC) [ Time Frame: Baseline up to 6 years ]
    Change from baseline in WHO FC according to non-invasive criteria will be assessed as per the low (I, II), intermediate (III), and high-risk category (IV).

  8. Change from Baseline in N-terminal-pro-hormone Brain Natriuretic Peptide (NT-proBNP) [ Time Frame: Baseline up to 6 years ]
    Change from baseline in NT-proBNP according to non-invasive criteria will be assessed as per the low (<300 nanogram per liters [ng/l]), intermediate (300-1400ng/l), and high-risk category (>1400 ng/l).

  9. Time to Worsening in WHO FC [ Time Frame: Up to 6 months ]
    Time to worsening in WHO FC will be reported.

  10. Change from Baseline in the Number of low-risk Noninvasive Criteria Based on WHO FC, 6MWD, and NT-proBNP [ Time Frame: Baseline up to 6 years ]
    Change from baseline in the number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP will be reported.

  11. Time to all-cause Death Based on the Number of low-risk Noninvasive criteria Based on WHO FC, 6MWD, and NT-proBNP [ Time Frame: Up to 6 years ]
    Time to all-cause death based on the number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP will be reported.

  12. Change from Baseline in Number of Participants Within Each Overall Risk Category (Low, Intermediate, or High) According to the Noninvasive Criteria [ Time Frame: Baseline up to 6 years ]
    Change from baseline in number of participants within each overall risk category (low, intermediate, or high) according to low-risk non-invasive criteria will be assessed as per the following parameters: WHO FC- low (I, II), intermediate (III), and high-risk category (IV); 6MWD- low (> 440m), intermediate (165-440m), and high-risk category (< 165m), and <165m; and NT-proBNP- low (<300ng/l), intermediate (300-1400ng/l), and high-risk category (>1400 ng/l).

  13. Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to Registry to Evaluate Early and Long-term PAH Disease Management (REVEAL) Lite 1 Variables [ Time Frame: Baseline up to 6 years ]
    Change from baseline in number of participants within each overall risk category (low, Intermediate, or High) according to REVEAL Lite 1 Variables will be reported. The REVEAL Lite 1 variables risk calculator determines the risk status, and the scores (ranges from 0 to 19) can be defined as: low risk as a score of less than or equal to (<=) 6, intermediate risk as a score of 7 or 8, and high risk as a score of greater than or equal to (>=) 9 for the survival rates.

  14. Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to REVEAL Lite 2 Variables [ Time Frame: Baseline up to 6 years ]
    Change from baseline in number of participants within each overall risk category (low, Intermediate, or High) according to REVEAL Lite 2 Variables will be reported. The REVEAL Lite 2 variables risk calculator determines the risk status, and the scores (ranges from 1 to 14) can be defined as: low risk as a score of <= 5, intermediate risk as a score of 6 or 7, and high risk as a score of >= 8 for the survival rates.

  15. Time to all-cause Death Based on the Risk Category Determined by the Noninvasive Criteria [ Time Frame: Up to 6 years ]
    Time to all-cause death based on the risk category determined by the noninvasive criteria will be reported.

  16. Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 1 [ Time Frame: Up to 6 years ]
    Time to all-cause death based on the risk category determined by the REVEAL Lite 1 will be reported.

  17. Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 2 [ Time Frame: Up to 6 years ]
    Time to all-cause death based on the risk category determined by the REVEAL Lite 2 will be reported.

  18. Risk Assessment Strategies for Clinical Worsening or Death [ Time Frame: Up to 6 years ]
    The assessment of risk category for clinical worsening and death will be assessed by the following 3 risk assessment strategies: number of low-risk noninvasive criteria based on WHO FC, 6MWD, and NT-proBNP.

  19. Change from Baseline in Health-related Quality of Life (HRQoL) as Measured by Symptoms and Impact Questionnaire for Use in Clinical Practice (SYMPACT-CP) Questionnaire [ Time Frame: Baseline up to 6 years ]
    A modified version of the PAH-SYMPACT for Use in Clinical Practice (SYMPACT-CP), has been created for use in routine clinical practice, as an assessment to support symptom monitoring and guide treatment decisions by healthcare providers. In the SYMPACT-CP, the 24-hour recall period for the oxygen use item and the 11 symptoms items have been modified to a 1-week recall period. The SYMPACT-CP also includes 11 impact items (with a 1-week recall period). This modification was made with the intention of administering the questionnaire at a single timepoint.

  20. Change from Baseline in Health Related Quality of Life Assessed by European Quality of Life (EuroQoL) Group, 5-Dimension, 5-Level Questionnaire (EQ-5D-5L) [ Time Frame: Baseline up to 6 years ]
    The EQ-5D-5L is an instrument to measure health-related quality of life consisting of a descriptive system and visual analogue scale (VAS). The descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels of perceived problems (1 indicating no problem, 2 indicating slight problems, 3 indicating moderate problems, 4 indicating severe problems, 5 indicating extreme problems). Participant selects an answer for each of 5 dimensions considering the response that best matches his or her health "today". The responses to the 5 dimensions are used to compute a single score ranging from 0 (worst health state) to 100 (better health state) representing the general health status of the individual.

  21. Change from Baseline in Health-Related Quality of life Status as Assessed by 36-item Short-Form Health Survey (SF-36) v2 Acute Questionnaire [ Time Frame: Baseline up to 6 years ]
    The SF-36 v2 acute questionnaire is a 36-item short form survey used to assess the participant's quality of life. In the SF-36 v2 Acute Questionnaire, participants are instructed to rate their health and capacity to perform activities of daily living in eight domains including physical functioning, physical role limitations, bodily pain, general health, vitality, social functioning, emotional role limitations, and mental health during the last week. The scores range from 0 (lowest or worst possible level of functioning) to 100 (highest or best possible level of functioning).

  22. Change from Baseline in PAH-specific Medication Adherence as Assessed by Morisky Medication Adherence Scale-8 (MMAS-8) Score [ Time Frame: Baseline up to 6 years ]
    The MMAS-8 is commonly used in standard clinical practice and a validated measure of treatment adherence. The MMAS-8 includes 8 questions assessing the extent of adherence or nonadherence and reasons for non-adherence.

  23. Change from Baseline in Medication Adherence Questions of each Prescribed PAH Therapy Class [ Time Frame: Baseline up to 6 years ]
    Change from baseline in medication adherence questions of each prescribed PAH therapy class will be reported.

  24. Change from Baseline in PAH-specific Medication Adherence as Assessed by Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire [ Time Frame: Baseline up to 6 years ]
    The PAH-SYMPACT questionnaire, applicable only for participants who enrolled in the PAH-SYMPACT substudy, is a PRO instrument and composed of 2 parts: the symptoms part and the Impacts part. There is no total symptom score; the 2 domains are: cardiopulmonary symptoms and cardiovascular symptoms. The Impacts part has 2 domains: Physical Impacts Domain and Cognitive/Emotional Impacts Domain which contain 7 and 4 items, respectively. Each item has 5-point Likert response scale (0=not at all, 1=mild, 2=moderate, 3=severe, and 4=very severe). The mean individual weekly symptom item scores are aggregated by domain, with the sum then being divided by the number of symptom items in the respective domain. This leads to the average weekly domain score ranging from 0-4 for each participant with higher scores indicating greater symptom severity or worse impact.

  25. Change from Baseline in Patient Global Assessment of Disease Severity (PGA-S) of PAH [ Time Frame: Baseline up to 6 years ]
    Change from baseline in PGA-S of PAH will be reported. It includes severity of PAH symptoms, such as, none, mild, moderate, severe and very severe.

  26. PAH Symptoms and Impact using SYMPACT-CP Questionnaire [ Time Frame: Baseline up to 6 years ]
    PAH symptoms and impact using SYMPACT-CP questionnaire will be reported.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Symptomatic pulmonary arterial hypertension (PAH) in any PAH subtype
  • PAH diagnosis confirmed by hemodynamic evaluation at rest at any time prior to or at the index date fulfilling all of the criteria below: a) Mean pulmonary artery pressure greater than (>) 20 millimeters of mercury (mm Hg), and b) Pulmonary artery wedge pressure or left ventricular end diastolic pressure less than or equal to (<=) 15 mm Hg, and c) Pulmonary vascular resistance greater than or equal to (>=) 3 Wood Units (that is, >= 240 dynes seconds per centimeters penta [dyn∙sec/cm^5])
  • Participant satisfies either a or b: a) Newly initiating 1 or more PAH therapy(ies) (as monotherapy or add-on therapy) at index date. These newly initiated PAH therapies should not have been used within 3 months of the index date; b) Taking macitentan 10 milligrams (mg) therapy (as monotherapy or in combination) with no changes in PAH therapy for within 3 months prior to the index date
  • All mandated assessments must be performed and recorded at the baseline visit before the initiation of the new PAH therapy at the index date or enrollment in the study.
  • For the pulmonary arterial hypertension-symptoms and impact (PAH-SYMPACT) substudy only: Participants initiating any endothelin receptor antagonist (ERA) or phosphodiesterase-5 inhibitor therapies at index date or at therapy change must provide consent to enroll in the optional PAH-SYMPACT substudy. Refusal to give consent for the optional PAH-SYMPACT substudy will not exclude a participant from participation in the main study

Exclusion Criteria:

  • Participants enrolled in any interventional clinical trial with an investigational therapy in the 3-month period prior to index date
  • Currently enrolled in an observational study sponsored or managed by a Janssen company
  • Presence of moderate or severe obstructive lung disease (forced expiratory volume in 1 second [FEV1] / forced vital capacity [FVC] <70%; and FEV1 <60% of predicted after bronchodilator administration) in participants with a known or suspected history of significant lung disease, as documented by a spirometry test performed within 1 year prior to screening
  • Presence of moderate or severe restrictive lung disease (for example, total lung capacity or FVC <60 percent [%] of normal predicted value) in participants with a known or suspected history of significant lung disease, as documented by a spirometry test performed within 1 year prior to screening

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04955990


Contacts
Layout table for location contacts
Contact: Study Contact 844-434-4210 Participate-In-This-Study@its.jnj.com

Locations
Show Show 100 study locations
Sponsors and Collaborators
Actelion
Investigators
Layout table for investigator information
Study Director: Actelion Clinical Trial Actelion
Layout table for additonal information
Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT04955990    
Other Study ID Numbers: CR109007
67896062PAH4005 ( Other Identifier: Actelion )
First Posted: July 9, 2021    Key Record Dates
Last Update Posted: July 20, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson and Johnson is available at www.janssen.com/clinical- trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) project site at yoda.yale.edu
URL: https://www.janssen.com/clinical-trials/transparency

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Pulmonary Arterial Hypertension
Familial Primary Pulmonary Hypertension
Hypertension
Vascular Diseases
Cardiovascular Diseases
Hypertension, Pulmonary
Lung Diseases
Respiratory Tract Diseases