Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil

Completed · Phase 3

Conditions studied: Primary Pulmonary Hypertension

In brief

The primary objective of this study is to evaluate the long-term safety and tolerability of LIQ861, a dry powder formulation of treprostinil, in patients with Pulmonary Arterial Hypertension (PAH).

Key facts

Study ID

NCT03399604

Run by

Liquidia Technologies, Inc.

People needed

121

Starts

2018-01-02

Expected to finish

2019-11-25

Last updated by the study team

2024-07-30

Who can join

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

You may qualify if…

• signed informed consent by patient prior to study enrollment
• 18 years of age or older
• If female of childbearing potential, a negative pregnancy test at the Baseline Visit and agrees to practice adequate birth control throughout the duration of the study. If the patient is postmenopausal or has documented surgical sterilization, a pregnancy test and birth control is not necessary.
• The patient has been diagnosed with PAH belonging to the following subgroups of the updated Nice Clinical Classification Group 1 (Simonneau, Gatzoulis et al. 2013), which include:
• Idiopathic PAH (1.1), or
• Heritable PAH (1.2), or
• Drug and toxin induced PAH (1.3), or
• PAH associated with connective tissue disease (1.4.1), HIV infection (1.4.2), or congenital heart disease (1.4.4) with simple systemic-to-pulmonary shunt at least 1 year after surgical repair
• The patient has been diagnosed with PAH and is NYHA Functional Class II - IV at Screening.
• has documented stable doses of approved inhaled therapy for at least 3 months prior to screening and is willing and able to transition from their prescribed dose of inhaled therapy to study drug, or
• has documented stable doses of no more than two approved oral therapies for at least 3 months prior to screening and is willing and able to add LIQ861 to their treatment regimen.
• The patient can complete a baseline six-minute walk distance (6MWD) ≥ 150 m.
• The patient has had evidence of FEV1 ≥ 60% and FEV1/FVC ratio ≥ 60% during the 6-month period prior to enrollment.

You may not qualify if…

• The patient's clinical condition is such that, in the opinion of the Investigator, they are not expected to remain clinically stable for the duration of the study.
• Patients with PH in the Updated Nice Classification Groups 2-5, or PAH Group 1 subgroups not covered by the inclusion criteria (e.g., associated with portal hypertension [1.4.3] or with schistosomiasis [1.4.5]).
• The patient is currently taking oral prostacyclin analogues or agonists, including treprostinil and selexipag.
• The patient has had any PAH medication (except for anticoagulants) discontinued within 14 days of Baseline.
• The patient has had a new type of chronic therapy (including but not limited to oxygen, a different class of vasodilator, diuretic, digoxin, and digitalis) for pulmonary hypertension added within 30 days of Baseline.
• The patient has uncontrolled systemic hypertension as evidenced by persistent systolic blood pressure greater than 160 mmHg or diastolic blood pressure greater than 100 mmHg.
• The patient has a history of hemodynamically significant left-sided heart disease including, but not limited to: aortic or mitral valve disease, pericardial constriction, restrictive or congestive cardiomyopathy, or coronary artery disease (CAD).
• The patient has had an atrial septostomy.
• The patient has any serious or life-threatening disease other than conditions associated with PAH (e.g. malignancy requiring aggressive chemotherapy, end stage renal disease, etc.).
• The patient is taking any excluded medications listed in the Investigator's Brochure, namely inhibitors and inducers of CYP2C8
• The patient has a hypersensitivity or allergy to any of the ingredients of LIQ861 or other clinically relevant allergies (clinical relevance per Investigator judgment).
• The patient has had a pulmonary infarction (defined as infarction in more than one lung segment documented by V/Q scan or pulmonary angiography) within two weeks of Screening.
• The patient has had a stroke or transient ischemic attack (TIA) within six months of Screening.
• The patient has evidence of an active uncontrolled sepsis or systemic infection during Screening.
• The patient is pregnant or lactating.
• The patient has any musculoskeletal disease or any other disease that would limit ambulation.
• The patient has participated in an investigational product or device study within the 30 days prior to Screening.
• The patient has current evidence of drug abuse in the opinion of the Investigator.
• The patient has severe hepatic impairment as evidenced by any history of ascites AND encephalopathy.
• The patient has severe renal impairment (eGFR < 35).
• The patient is taking inhaled treprostinil doses of greater than 90 μg (more than 15 breaths).
• Additional Exclusion Criteria for PK Sub-Study:
• The patient meets any of Primary Exclusion Criteria #1 - 19.
• The patient has moderate or severe renal impairment (eGFR < 60).
• The patient is taking inhaled treprostinil doses of greater than 72 μg (more than 12 breaths).

Where it is running

• Banner University Medical Center — Phoenix, Arizona, United States
• Arizona Pulmonary Specialists, Ltd. — Phoenix, Arizona, United States
• West Los Angeles VA Healthcare Center — Los Angeles, California, United States
• UC Davis Medical Center — Sacramento, California, United States
• Los Angeles Biomedical Research Center — Torrance, California, United States
• University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States
• University of Florida — Gainesville, Florida, United States
• Mayo Clinic-Jacksonville — Jacksonville, Florida, United States
• AdventHealth — Orlando, Florida, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• Wellstar Research Institute — Marietta, Georgia, United States
• Northwestern Medicine, Feinberg School of Medicine — Chicago, Illinois, United States
• University of Chicago Medicine — Chicago, Illinois, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Kentuckiana Pulmonary Research Center — Louisville, Kentucky, United States
• Ochsner Medical Center — New Orleans, Louisiana, United States
• Tufts Medical Center — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Mayo Clinic-Rochester — Rochester, Minnesota, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• University of New Mexico Health Science Center — Albuquerque, New Mexico, United States
• NYU Winthrop University Hospital — Mineola, New York, United States
• NYU Langone Health — New York, New York, United States
• University of North Carolina School of Medicine — Chapel Hill, North Carolina, United States
• University of Cincinnati Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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