Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Dry Powder Inhalation of Treprostinil

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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Primary Pulmonary Hypertension
• Intervention / Treatment: Drug: LIQ861 Inhaled Treprostinil

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).

• Study Type: Interventional
• Enrollment (Actual): 92
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Banner University Medical Center
    • Phoenix, Arizona, United States, 85012
      • Arizona Pulmonary Specialists, Ltd.
  • California
    • Los Angeles, California, United States, 90073
      • West Los Angeles VA Healthcare Center
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center
    • Torrance, California, United States, 90502
      • Los Angeles Biomedical Research Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz Medical Campus
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic-Jacksonville
    • Orlando, Florida, United States, 32803
      • AdventHealth
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University School Of Medicine
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Medicine
    • Chicago, Illinois, United States, 60611
      • Northwestern Medicine, Feinberg School of Medicine
  • Kansas
    • Kansas City, Kansas, United States, 66103
      • University of Kansas Medical Center
  • Louisiana
    • New Orleans, Louisiana, United States, 70121
      • Ochsner Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Tufts Medical Center
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic-Rochester
  • New Mexico
    • Albuquerque, New Mexico, United States, 87106
      • University of New Mexico Health Science Center
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • University of Cincinnati Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science Center
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • UPMC Presbyterian Hospital
    • Pittsburgh, Pennsylvania, United States, 15212
      • Alleghany General Hospital
  • Texas
    • Dallas, Texas, United States, 75390
      • UT Southwestern Medical Center
    • Houston, Texas, United States, 77030
      • Houston Methodist Lung Center
    • San Antonio, Texas, United States, 78229
      • University of Texas Health Science Center at San Antonio
  • Virginia
    • Falls Church, Virginia, United States, 22042
      • Inova Fairfax Medical Campus

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: LIQ861 Inhaled Treprostinil; LIQ861 inhaled treprostinil at capsule strengths of 25 μg, 50 μg, 75 μg and 100 μg. LIQ861 will be administered using the RS00 Model 8 dry powder inhalation (DPI) device (Plastiape S.p.A.; Osnago, Italy) at dose levels of 25 μg capsule strength to 200 μg capsule strength treprostinil four times a day (QID) in individual patients. Titrating to dose levels beyond 200 μg capsule strength QID, under clinical investigator supervision, requires review and approval from the Medical Monitor.

Drug: LIQ861 Inhaled Treprostinil; LIQ861 bulk powder is generated from a treprostinil/excipient matrix from which particles of precise size and shape are created and filled into a hydroxypropyl methylcellulose (HPMC) capsule (size 3). LIQ861 capsules are provided in capsule strengths of 25 μg, 50 μg, 75 μg and 100 μg treprostinil.; Other Names: Inhaled treprostinil; Inhaled prostacyclin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment emergent adverse events (AEs).	Treatment-emergent adverse events and serious adverse events will be grouped by MedDRA System Organ Class, dose level at onset, time on drug at onset, and relationship to dose titration.	Baseline until the end of study, approximately 2.5 years (Dec-2021).

Collaborators and Investigators

• Sponsor: Liquidia Technologies, Inc.
• Collaborators: Nuventra, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 18, 2019
• Primary Completion (Anticipated): December 31, 2021
• Study Completion (Anticipated): March 30, 2022

Study Registration Dates

• First Submitted: June 5, 2019
• First Submitted That Met QC Criteria: June 18, 2019
• First Posted (Actual): June 20, 2019

Study Record Updates

• Last Update Posted (Actual): August 30, 2021
• Last Update Submitted That Met QC Criteria: August 26, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• 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
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension; Pulmonary Arterial Hypertension; Familial Primary Pulmonary Hypertension; Hypertension, Pulmonary; Antihypertensive Agents; Treprostinil
• Other Study ID Numbers: LTI-302

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No