Safety and Efficacy of Inhaled Treprostinil in Adult PH With ILD Including CPFE

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Pulmonary Hypertension, Interstitial Lung Disease, Combined Pulmonary Fibrosis and Emphysema

In brief

This was a multicenter, randomized (1:1 inhaled treprostinil: placebo), double-blinded, placebo-controlled trial to evaluate the safety and efficacy of inhaled treprostinil in subjects with pre-capillary pulmonary hypertension (PH) associated with interstitial lung disease (ILD) including combined pulmonary fibrosis and emphysema (CPFE). The study included 326 patients at approximately 120 clinical trial centers. The treatment phase of the study lasted approximately 16 weeks. Patients who completed all required assessments were eligible to enter an open-label, extension study (RIN-PH-202).

Key facts

Study ID

NCT02630316

Run by

United Therapeutics

People needed

326

Starts

2017-02-03

Expected to finish

2019-12-26

Last updated by the study team

2022-07-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subject voluntarily gave informed consent to participate in the study.
• Males and females aged 18 years or older at the time of informed consent.
• a. Females of reproductive potential were non-pregnant (as confirmed by a urine pregnancy test at screening) and non-lactating, and: i. Abstained from intercourse (when in line with their preferred and usual lifestyle), or ii. Used 2 medically acceptable, highly effective forms of contraception for the duration of study, and at least 30 days after discontinuing study drug.
• b. Males with a partner of childbearing potential used condoms for the duration of treatment and for at least 48 hours after discontinuing study drug.
• The subject had a confirmed diagnosis of WHO Group 3 PH based on computed tomography (CT) imaging which was performed within 6 months prior to randomization and demonstrated evidence of diffuse parenchymal lung disease. Subjects had any form of ILD or CPFE.
• Subjects were required to have a right heart catheterization (RHC) within 1 year prior to randomization with the following documented parameters:
• Pulmonary vascular resistance (PVR) >3 Wood Units (WU) and
• A pulmonary capillary wedge pressure (PCWP) of <15 mmHg and
• A mean pulmonary arterial pressure (mPAP) of >25 mmHg
• Baseline 6MWD ≥100 m.
• Subjects on a chronic medication for underlying lung disease (ie, pirfenidone, nintedanib, etc) were on a stable and optimized dose for ≥30 days prior to randomization.
• In the opinion of the Investigator, the subject was able to communicate effectively with study personnel, and was considered reliable, willing and likely to be cooperative with protocol requirements, including attending all study visits.
• Subjects with connective tissue disease (CTD) had a Baseline forced vital capacity (FVC) of <70%.

You may not qualify if…

• The subject had a diagnosis of PAH or PH for reasons other than WHO Group 3 PH ILD as outlined in Inclusion Criterion 3.
• The subject showed intolerance or significant lack of efficacy to a prostacyclin or prostacyclin analogue that resulted in discontinuation or inability to effectively titrate that therapy.
• The subject received any PAH-approved therapy including: prostacyclin therapy (ie, epoprostenol, treprostinil, iloprost, or beraprost; except for acute vasoreactivity testing), prostacyclin (IP) receptor agonist (selexipag), endothelin receptor antagonist (ERA), phosphodiesterase type 5 inhibitor (PDE5-I), or soluble guanylate cyclase (sGC) stimulator within 60 days of randomization.
• The subject had evidence of clinically significant left-sided heart disease as defined by:
• PCWP >15 mmHg
• Left ventricular ejection fraction <40%. Note: Subjects with abnormal left ventricular function attributable entirely to impaired left ventricular filling due to the effects of right ventricular overload (ie, right ventricular hypertrophy and/or dilatation) were not excluded.
• The subject was receiving >10 L/min of oxygen supplementation by any mode of delivery at rest at Baseline.
• Current use of any inhaled tobacco/marijuana products or significant history of drug abuse at the time of informed consent.
• Exacerbation of underlying lung disease or active pulmonary or upper respiratory infection within 30 days of randomization.
• Initiation of pulmonary rehabilitation within 12 weeks prior to randomization.
• In the opinion of the Investigator, the subject had any condition that would interfere with the interpretation of study assessments or has any disease or condition (ie, peripheral vascular disease, musculoskeletal disorder, morbid obesity) that would likely be the primary limit to ambulation (as opposed to PH).
• Use of any investigational drug/device, or participation in any investigational study with therapeutic intent within 30 days prior to randomization.
• Severe concomitant illness limiting life expectancy (<6 months).
• Acute pulmonary embolism within 90 days of randomization.

Where it is running

• IMC-Diagnostic & Medical Clinic — Mobile, Alabama, United States
• Arizona Pulmonary Specialists, Ltd. — Phoenix, Arizona, United States
• St. Joseph's Hospital and Medical Center — Phoenix, Arizona, United States
• University of Arizona — Tucson, Arizona, United States
• Cedars-Sinai Medical Center, Advanced Health Sciences Pavilion — Beverly Hills, California, United States
• University of California San Francisco - Fresno — Fresno, California, United States
• University of California San Diego — La Jolla, California, United States
• VA Long Beach Healthcare System — Long Beach, California, United States
• University of Southern California Health Sciences — Los Angeles, California, United States
• Department of Veterans Affairs Greater Los Angeles Healthcare System — Los Angeles, California, United States
• Pacific Pulmonary Medical Group — Riverside, California, United States
• Kaiser Permanente - Roseville — Roseville, California, United States
• University of California Davis Medical Center — Sacramento, California, United States
• Kaiser Permanente — San Francisco, California, United States
• University of Colorado Hospital - Cardiac and Vascular Center — Aurora, Colorado, United States
• National Jewish Health — Denver, Colorado, United States
• Yale New Haven Hospital — New Haven, Connecticut, United States
• Medstar Georgetown University Hospital — Washington D.C., District of Columbia, United States
• MedStar Washington Hospital Center — Washington D.C., District of Columbia, United States
• Florida Lung, Asthma & Sleep Specialists, P.A. — Celebration, Florida, United States
• St. Francis Sleep, Allergy and Lung Institute — Clearwater, Florida, United States
• University of Florida Clinical Research Center — Gainesville, Florida, United States
• University of Florida College of Medicine, Jacksonville — Jacksonville, Florida, United States
• Mayo Clinic Jacksonville — Jacksonville, Florida, United States
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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