Clinical trial NCT03992755: Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Dry Powder Inhalation of Treprostinil
Official title: A Global, Open-Label Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Inhaled LIQ861 (Treprostinil) in Pulmonary Arterial Hypertension (WHO Group 1) Patients
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Primary Pulmonary Hypertension
Keywords: Pulmonary Arterial Hypertension
Idiopathic Pulmonary Arterial Hypertension
Heritable Pulmonary Arterial Hypertension
Drug Induced Pulmonary Arterial Hypertension
Toxin Induced Pulmonary Arterial Hypertension
Connective tissue disease
Lead sponsor: Liquidia Technologies, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Nuventra, Inc.
First posted: 2019-06-20
Enrollment: 92
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: The study will evaluate the long-term safety of LIQ861 in PAH (WHO Group 1) patients who have completed a Liquidia LIQ861 clinical study. All patients will be treated on an outpatient basis until regulatory approval of LIQ861 or study is terminated by sponsor. Study enrollment will occur after final assessments from a prior study have been completed.
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to evaluate the long-term safety of LIQ861 in patients with pulmonary arterial hypertension (PAH).

Detailed description: One of the greatest impediments to patient treatment satisfaction with current inhaled treprostinil therapy is inconvenience. Currently, PAH patients using inhaled treprostinil may require more than 36 breaths per day using a nebulizer requiring daily set up and cleaning. The use of a discrete, hand-held dry powder inhaler to deliver treprostinil to the lungs could represent a major improvement in convenience and patient satisfaction, thereby improving the quality of life for PAH patients. Liquidia is pursuing approval of LIQ861, an inhalation dry powder formulation of treprostinil that is produced using Liquidia's PRINT® Technology (Particle Replication in Nonwetting Templates), as an alternative to current inhaled treprostinil therapy for the treatment of patients with PAH (WHO Group 1).

Eligibility criteria: Inclusion Criteria:

1. Evidence of a personally signed and dated informed consent document exists indicating that the patient has been informed of all pertinent aspects of the study prior to initiation of any study-related procedures.
2. Patient is willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
3. Patient has fulfilled all entry criteria at the time of enrollment in original study with LIQ861.
4. Patient has completed the protocol defined end of study procedures or met a protocol-defined and adjudicated endpoint in the original LIQ861 study in which they were enrolled.
5. Patient, whether male or female, agrees to use a medically acceptable method of contraception throughout the entire study period from informed consent through the termination visit, if the possibility of conception exists. Eligible male and female patients must also agree not to participate in a conception process (e.g., actively attempt to become pregnant or to impregnate, sperm donation, in vitro fertilization) during the study and for 30 days after the last dose of LIQ861.

Exclusion Criteria:

1. Patient prematurely discontinued LIQ861 due to a drug-related AE/SAE or tolerability issue in the original LIQ861 study in which they were enrolled, or patient did not complete protocol defined study procedures at an end of study visit (not Early Termination visit) in their original LIQ861 study.
2. Patient withdrew consent during participation in another LIQ861 study.
3. Patient is a female who wishes to become pregnant or who has a positive pregnancy test on Day 1 (LTI-302 Study Initiation Visit).
4. Patient has undergone lung or heart/lung transplant or the initiation of parenteral (intravenous \[IV\] infusion or subcutaneous injection) therapy with a prostacyclin during the time since participation in their original LIQ861 study.
5. Any reason exists that, in the opinion of the Investigator or Medical Monitor, precludes the patient from participating in the study, e.g., any previous or intercurrent medical condition that may increase the risk associated with study participation or that would confound study analysis or impair study participation or cooperation.

Primary outcomes:
measure: Number of participants with treatment emergent adverse events (AEs).
timeFrame: Baseline until the end of study, approximately 2.5 years (Dec-2021).
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Banner University Medical Center
state: Arizona
city: Phoenix
country: United States
facility: Arizona Pulmonary Specialists, Ltd.
state: Arizona
city: Los Angeles
country: United States
facility: West Los Angeles VA Healthcare Center
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Torrance
country: United States
facility: Los Angeles Biomedical Research Center
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic-Jacksonville
state: Florida
city: Orlando
country: United States
facility: AdventHealth
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Chicago
country: United States
facility: Northwestern Medicine, Feinberg School of Medicine
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medicine
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: New Orleans
country: United States
facility: Ochsner Medical Center
state: Louisiana
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic-Rochester
state: Minnesota
city: Albuquerque
country: United States
facility: University of New Mexico Health Science Center
state: New Mexico
city: Cincinnati
country: United States
facility: University of Cincinnati Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science Center
state: Oregon
city: Pittsburgh
country: United States
facility: Alleghany General Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Presbyterian Hospital
state: Pennsylvania
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Houston Methodist Lung Center
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Science Center at San Antonio
state: Texas
city: Falls Church
country: United States
facility: INOVA Fairfax Medical Campus
state: Virginia
Study officials:
affiliation: Tufts Medical Center
name: Nicholas S Hill, MD
role: PRINCIPAL_INVESTIGATOR
References: