Patient Global Impression Questions for Activity-Induced Symptoms in Participants With PAH (PRN)

United Therapeutics

Status

Completed

Conditions

Pulmonary Arterial Hypertension

Treatments

Drug: Treprostinil

Drug: Non-Treprostinil PAH Medications

Study type

Observational

Funder types

Industry

Identifiers

NCT03888365

RIN-PRN-201

Details and patient eligibility

About

This is an observational, multicenter, single-day, Phase 2 study. This study will include a 14-day Screening Period and Study Day 1 clinic visit. Participants will be required to perform an activity to induce symptoms of PAH, and participants' severity of self-reported symptoms of PAH will be measured from pre-activity, immediately after the activity, and through the 30-minute recovery. Participants will be asked about their PAH symptoms using 3 PGI-S questions that address their overall PAH symptoms, shortness of breath, and physical fatigue.

Full description

This is an observational, multicenter, single-day, Phase 2 study. This study will include a 14-day Screening Period and Study Day 1 clinic visit. Participants will be required to perform an activity to induce symptoms of PAH, and participants' severity of self-reported symptoms of PAH will be measured from pre-activity, immediately after the activity, and through the 30-minute recovery. Participants will be asked about their PAH symptoms using 3 Patient Global Impression of Severity (PGI-S) questions that address their overall PAH symptoms, shortness of breath, and physical fatigue.

PAH symptoms will be induced via the Incremental Shuttle Walk Test (ISWT). The ISWT used in this study required the participant to walk back and forth on a 10-meter course. The total number of shuttles completed by a participant during the Screening ISWT will be the maximum targeted for that participant during the remaining ISWTs in the study.

After Screening, participants will be assigned to 1 of 2 cohorts based on PAH medications as prescribed by their physician: Cohort A will include participants who are currently prescribed and using inhaled treprostinil for the treatment of PAH and Cohort B will include participants who are taking other PAH medications (instead of inhaled treprostinil).

The study also includes 2 periods. One period for participants in Cohort A (Treprostinil Users), included an ISWT initiated within 30 minutes of the previous dose (expected peak level) and the other period included an ISWT within 3 to 4 hours of the previous dose of inhaled treprostinil (expected trough level). Participants in Cohort B (Non-Treprostinil Users), an ISWT will be initiated approximately 4 hours after the morning dose of PAH medication (Period 1) and an ISWT initiated at least 1 hour following completion of the previous ISWT (Period 2). Participants will be provided at least a 1-hour period for rest between ISWTs (until participant feels they are rested enough to perform again at their baseline level) prior to Period 2 assessments.

Enrollment

43 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Participant voluntarily gives informed consent to participate in the study.
Males and females aged 18 years and above at the time of informed consent.
Established primary diagnosis of PAH that is either idiopathic or familial PAH (WHO Group 1), collagen vascular disease associated PAH, PAH associated with HIV infection, PAH induced by anorexigens/toxins, or PAH associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥1 years).
Participant is deemed WHO Functional Class 1, 2, or 3.
Participant has shortness of breath upon exertion (exhibits a ≥1-point change in Borg dyspnea score) as assessed by the ISWT and a minimum completion of 3 shuttles (30 meters) of the ISWT. Participant may have other symptoms as well.
Participant is on stable dose of all FDA-approved PAH treatments (exceptions are anticoagulants and diuretics) for at least 60 days prior to Screening.
In the opinion of the Investigator, the participant can communicate effectively with study personnel, and is considered reliable, willing, and likely to be cooperative with protocol requirements.

Exclusion criteria

The participant is known to be pregnant or nursing.
The participant has PAH related to any condition not covered under inclusion criteria, including, but not limited to, pulmonary venous hypertension, pulmonary venoocclusive disease, pulmonary capillary hemangiomatosis, chronic thromboembolic pulmonary hypertension, or other conditions under WHO Group 2, 3, 4, and 5 classifications.
The participant has evidence of clinically significant left-sided heart disease (including, but not limited to, left ventricular ejection fraction <40%, left ventricular hypertrophy) or clinically significant cardiologic conditions, such as congestive heart failure, coronary artery disease, or valvular heart disease.
The participant has any form of congenital heart disease (repaired or unrepaired; other than a patent foramen ovale).
The participant has any ambulatory or orthopedic limitations that would interfere with the ability to perform the activity.
The participant has been hospitalized within 30 days of Screening.
Current use of prostacyclin analogs/agonists, except inhaled treprostinil, for the treatment of PAH.
Use of any other investigational drug/device, or participation in any investigational study with therapeutic intent within 30 days of Screening (concurrent participation in registry studies is allowed).
Any other clinically significant illness that, in the opinion of the Investigator, might put the participant at risk of harm during the study or might adversely affect the interpretation of the study data.