This was a Phase 1b safety and tolerability single-sequence study in which PAH subjects on a stable regimen of Tyvaso switched to a corresponding dose of TreT.
United Therapeutics Corporation (UTC) developed a combination drug-device product comprised of a dry powder formulation of Treprostinil Inhalation Powder (TreT) and a small, portable, dry powder inhaler. In this Phase 1b safety and tolerability study, patients with PAH on a stable dose of Tyvaso (6 to 12 breaths 4 times daily \[QID\]) were evaluated after switching to a corresponding dose of TreT. Patients underwent PK assessments, safety assessments, a 6-Minute Walk Test (6MWT), and questionnaires for satisfaction/preference for inhaled devices and patient-reported PAH symptoms and impact. Following 3 weeks of treatment with TreT, patients were offered the opportunity to participate in the Optional Extension Phase until the drug/device became commercially available.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
51
Treprostinil inhalation powder single-use cartridges containing either 32 or 48 micrograms of treprostinil per cartridge (QID)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Department of Veterans Affairs Greater Los Angeles Healthcare System
Los Angeles, California, United States
Change in 6-Minute Walk Distance (6MWD) From Baseline to Week 3
6MWD was evaluated at study entry and after 3 weeks of treatment with TreT.
Time frame: From Baseline to 3 weeks of treatment with TreT
Subject Satisfaction With and Preference for Inhaled Treprostinil Devices
Subject satisfaction with and preference for the inhaled treprostinil device was evaluated with the Preference Questionnaire for Inhaled Treprostinil Devices (PQ-ITD). The PQ-ITD is a questionnaire given to evaluate subject satisfaction with and preference for inhaled treprostinil devices. The questionnaire provides 12 different statements around inhaled device satisfaction and allows for 5 response options: strongly disagree, disagree, neutral, agree, and strongly agree.
Time frame: After 3 weeks of treatment with TreT (after switching from the Tyvaso Inhalation System)
Change in Patient-reported PAH Symptoms and Impact From Baseline to Week 3
Patient-reported PAH symptoms and impact were evaluated with the PAH Symptoms and Impact (PAH-SYMPACT) Questionnaire. The PAH-SYMPACT is a 23-item patient-reported outcome questionnaire that consists of 11 symptom items, 11 impact items, and 1 item on oxygen use. The symptom items are divided into cardiopulmonary and cardiovascular domains, and the impact items are divided into physical and emotional/cognitive domains. Symptom and impact domain scores (range 0 to 4) are calculated as the sum of the scores for the items included in the domain divided by the number of items in the domain. For all domains, a higher score indicates more severe symptoms/impacts.
Time frame: From Baseline to 3 weeks of treatment with TreT
Change in Patient-reported PAH Symptoms and Impact From Baseline to Week 11 (for Subjects Participating in the OEP)
Patient-reported PAH symptoms and impact were evaluated with the PAH Symptoms and Impact (PAH-SYMPACT) Questionnaire. The PAH-SYMPACT is a 23-item patient-reported outcome questionnaire that consists of 11 symptom items, 11 impact items, and 1 item on oxygen use. The symptom items are divided into cardiopulmonary and cardiovascular domains, and the impact items are divided into physical and emotional/cognitive domains. Symptom and impact domain scores (range 0 to 4) are calculated as the sum of the scores for the items included in the domain divided by the number of items in the domain. For all domains, a higher score indicates more severe symptoms/impacts.
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Ascension / St. Vincent's Lung Institute
Jacksonville, Florida, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of South Florida Center for Advanced Lung Disease
Tampa, Florida, United States
Cleveland Clinic Florida
Weston, Florida, United States
Emory University Hospital
Atlanta, Georgia, United States
The University of Kansas Medical Center
Kansas City, Kansas, United States
University of Louisville Clinical Trials Unit
Louisville, Kentucky, United States
Ochsner Medical Center
New Orleans, Louisiana, United States
...and 6 more locations
Time frame: From Baseline to 11 weeks of treatment with TreT