N/A
N=43
Patient Global Impression Questions for Activity-Induced Symptoms in Participants With PAH
Pulmonary Arterial Hypertension
Bottom Line
View on ClinicalTrials.gov: NCT03888365 ↗Enrolled (actual)
43
Serious AEs
0.0%
Results posted
Mar 2021
Primary outcome: Primary: Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1 — 1.25; 1.65; 1.15; 1.90 scores on a scale
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Treprostinil (Drug); Non-Treprostinil PAH Medications (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- United Therapeutics
- Primary completion
- Sep 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1 |
1.25; 1.65; 1.15; 1.90; 1.30; 1.40 | — |
| PRIMARY Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1 |
1.10; 1.75; 1.10; 1.85; 1.25; 1.50 | — |
| PRIMARY Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1 |
1.22; 1.74; 1.17; 1.78; 1.17; 1.61 | — |
| PRIMARY Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1 |
1.13; 1.87; 1.04; 2.13; 1.13; 1.91 | — |
| SECONDARY Change From Baseline in Modified Borg Dyspnea Scores at Day 1 |
0.28; 3.00; 0.23; 3.13; 0.24; 2.85 | — |
Summary
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT03888365). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.