An Open-Label ProSpective MultiCENTer Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in PH (ASCENT)

An Open-Label ProSpective MultiCENTer Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in Pulmonary Hypertension

Study LTI-401 is an open-label, multicenter study which will evaluate the safety and tolerability of LIQ861 in subjects who have WHO Group 1 & 3 PH.

Study Overview

• Status: Recruiting
• Conditions: Pulmonary Hypertension; Interstitial Lung Disease
• Intervention / Treatment: Drug: LIQ861

Detailed Description

Open-label, multicenter study which will evaluate the safety and tolerability of LIQ861 in subjects who have WHO Group 1 & 3 PH.

Cohort A will include approximately 60 subjects who have WHO Group 3 Pulmonary Hypertension associated with interstitial lung disease (PH-ILD)

Cohort B will include approximately 20 subjects who have WHO Group 3 PH-ILD, have been on protocol specified dosing of inhaled treprostinil QID, not at treatment goal, and able to transition to LIQ861.

The primary objective of this study is to evaluate the safety and tolerability of LIQ861 in subjects with WHO Group 1 & 3 Pulmonary Hypertension (PH).

• Study Type: Observational
• Enrollment (Estimated): 80

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85012
      • Recruiting
      • Arizona Pulmonary Specialists
      • Contact: Akshay Muraldihar; Phone Number: 602-271-0832; Email: nidhi@azpulm.com
  • California
    • Fresno, California, United States, 93720
      • Recruiting
      • The Medical Research Group, Inc.
      • Contact: Vijay Balasubramanian; Phone Number: (559) 367-3095; Email: mmoreno@themedicalresearchgroup.com
    • La Jolla, California, United States, 92093
      • Withdrawn
      • University of California San Diego
    • Los Angeles, California, United States, 90095
      • Active, not recruiting
      • UCLA Westwood
    • Sacremento, California, United States, 95817
      • Active, not recruiting
      • UC Davis
    • San Francisco, California, United States, 94143
      • Active, not recruiting
      • UCSF
  • Colorado
    • Denver, Colorado, United States, 80206
      • Recruiting
      • National Jewish Health
      • Contact: Patty George; Phone Number: 303-398-1233; Email: henriksenj@njhealth.org
  • Connecticut
    • Hartford, Connecticut, United States, 06102
      • Withdrawn
      • Hartford Hospital
  • Florida
    • Orlando, Florida, United States, 32803
      • Recruiting
      • Advent Health
      • Contact: James Tarver; Phone Number: 407-303-9984; Email: Tracey.Purvis@AdventHealth.com
    • Tampa, Florida, United States, 33606
      • Recruiting
      • Tampa General Hospital Center of Research Excellence
      • Contact: Ricardo Restrepo; Phone Number: 215-964-7399; Email: ricardo@health.usf.edu
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Recruiting
      • Vincent Medical Group
      • Contact: Ashwin Ravichandran; Phone Number: 317-338-8042; Email: ashwin.ravichandran@ascension.org
  • Kentucky
    • Louisville, Kentucky, United States, 40241
      • Withdrawn
      • Norton Pulmonary Specialists
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Withdrawn
      • Tufts Medical Center
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Active, not recruiting
      • University of Michigan
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Active, not recruiting
      • University of Minnesota
  • New York
    • New York, New York, United States, 10016
      • Active, not recruiting
      • NYU Langone Medical Center
    • Rochester, New York, United States, 14627
      • Active, not recruiting
      • University of Rochester
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • Withdrawn
      • University of North Carolina at Chapel Hill
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • Active, not recruiting
      • University of Cincinnati Health
  • Oregon
    • Bend, Oregon, United States, 97701
      • Recruiting
      • Summit Health
      • Contact: Jeremy Feldman; Phone Number: 541-382-2811; Email: jfeldman@summithealth.com
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina
      • Contact: Rachana Krishna; Phone Number: 843-792-3711; Email: millare@musc.edu
  • Texas
    • Houston, Texas, United States, 77030
      • Active, not recruiting
      • Houston Methodist
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • Active, not recruiting
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Subject with PH-ILD

Description

Cohort A Key Inclusion Criteria:

1. Males or Females between 18 years to 80 years of age.

2. Has a confirmed diagnosis of WHO Group 3 PH-ILD based on CT chest imaging performed, which demonstrates evidence of diffuse parenchymal lung disease and FEV1/FVC (absolute values) ≥ 70% and are required to have evidence of pulmonary hypertension (PH) as demonstrated from right heart catheterization (RHC) with the following hemodynamic parameters.

   1. i) Pulmonary vascular resistance (PVR) ≥ 3 Wood Units (WU) and ii) Pulmonary capillary wedge pressure (PCWP) of ≤ 15 mmHg and iii) A mean pulmonary arterial pressure (mPAP) of ≥ 30 mmHg.

      OR

   2. An exploratory subset of subjects with ILD: i) Pulmonary vascular resistance (PVR) ≥ 3 Wood Units (WU) and ii) Pulmonary capillary wedge pressure (PCWP) of ≤ 15 mmHg and iii) A mean pulmonary arterial pressure (mPAP) of ≥ 21 mmHg.
3. 6-minute walk distance of ≥ 125 meters

Cohort A Key Exclusion Criteria:

A Subject is not eligible for inclusion in the study if any of the following criteria apply:

1. PH in the Updated WHO Classification Groups 1, 2, 4, or 5.
2. History of hemodynamically significant left-sided heart disease.
3. Exacerbation of underlying lung disease or active pulmonary or upper respiratory infections.
4. Initiation of pulmonary rehabilitation.

Cohort B Key Inclusion Criteria

1. Male or Females between 18 years to 75 years of age at Screening.

2. Has a diagnosis of WHO Group 3 PH-ILD confirmed with CT chest imaging performed at the screening visit, which demonstrates evidence of diffuse parenchymal lung disease and FEV1/FVC (absolute values) ≥70%. Subjects are required to have evidence of pulmonary hypertension (PH) as demonstrated from right heart catheterization (RHC) with the following documented parameters:

   1. Pulmonary vascular resistance (PVR) ≥3 Wood Units (WU) and
   2. Pulmonary capillary wedge pressure (PCWP) of ≤ 15 mmHg and
   3. A mean pulmonary arterial pressure (mPAP) of ≥ 25 mmHg
3. Subjects must be on protocol specified dose of inhaled treprostinil QID not at treatment goal, and able to transition from their prescribed dose of inhaled Treprostinil therapy to LIQ861.
4. 6-minute walk distance of ≥ 200 meters

Cohort B Key Exclusion Criteria

1. PH in the Updated WHO Classification Groups 1, 2, 4, or 5.
2. Inability to titrate inhaled treprostinil above 5 breaths (Tyvaso®) or above 16 mcg Tyvaso DPI®.
3. History of Bronchospasm with Tyvaso or Tyvaso DPI.
4. History of persistent moderate asthma or severe asthma.
5. History of hemodynamically significant left-sided heart disease.
6. Exacerbation of underlying lung disease or active pulmonary or upper respiratory infections.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort A; PH-ILD	Drug: LIQ861; trepostinil inhalation powder in combination with inhaler
Cohort B; PH-ILD	Drug: LIQ861; trepostinil inhalation powder in combination with inhaler

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cohort A: Incident of treatment-emergent drug/device-related adverse events and Serious Adverse Events (SAEs)	Treatment-emergent AEs and SAEs will be grouped by MedDRA System Organ Class, dose level at onset, time on drug at onset, and relationship to dose titration. Device-related AEs and SAEs 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 the study.
Cohort B: Number of participants with treatment-emergent drug/device-related adverse events and Serious Adverse Events (SAEs)	Treatment-emergent AEs and SAEs will be grouped by MedDRA System Organ Class, dose level at onset, time on drug at onset, and relationship to dose titration. Device-related AEs and SAEs will be grouped by MedDRA System Organ Class, dose level at onset, time on drug at onset, and relationship to dose titration.	16 weeks

Collaborators and Investigators

• Sponsor: Liquidia Technologies, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 28, 2023
• Primary Completion (Estimated): July 31, 2027
• Study Completion (Estimated): February 8, 2028

Study Registration Dates

• First Submitted: November 6, 2023
• First Submitted That Met QC Criteria: November 10, 2023
• First Posted (Actual): November 13, 2023

Study Record Updates

• Last Update Posted (Actual): June 26, 2026
• Last Update Submitted That Met QC Criteria: June 24, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension; Lung Diseases, Interstitial; Hypertension, Pulmonary
• Other Study ID Numbers: LTI-401

Drug and device information, study documents

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