A Phase 3 Study to Evaluate the Safety and Tolerability of L606 in Subjects With PAH or PH-ILD

NCT04691154 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: PBI L606p3
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 8

Brief Summary

This Phase 3, 2-part, open-label, multicenter study aims to demonstrate the safety and tolerability of L606 in patients with PAH or PH-ILD. The study will determine the short-term and long-term safety and tolerability of L606 in this patient population.

Conditions

Pulmonary Arterial Hypertension; Pulmonary Hypertension Due to Lung Diseases

Keywords

treprostinil; Pulmonary Arterial Hypertension; inhalation; Pulmonary Hypertension Due to Lung Diseases

Outcome Measures

Primary Outcomes (3)

• Safety/Tolerability assessed by incidence of treatment-emergent AEs/SAEs

  (MSP for Cohort A): Proportion of patients with PAH or PH-ILD on a stable Tyvaso dose who would develop treatment-emergent AEs/SAEs after switching to L606 dosing for up to 2 weeks during MSP.

  2 weeks

• Safety/Tolerability assessed by incidence of treatment-emergent AEs/SAEs

  (MSP for Cohort B): Proportion of patients with PAH (not initially on prostacyclin therapy) who would develop treatment-emergent AEs/SAEs during 12 weeks of the MSP with titration on twice daily L606.

  12 weeks

• Safety/Tolerability assessed by incidence of treatment-emergent AEs/SAEs (long-term)

  (OEP for Cohorts A and B): Proportion of subjects with PAH or PH-ILD, choosing to continue twice daily L606 dosing for up to 48 weeks, who would develop treatment emergent AEs/SAEs up to 48 hours after the last dose.

  48 weeks

Study Arms (1)

L606

EXPERIMENTAL

Combination Product: L606 inhalation suspension

Interventions

L606 inhalation suspension COMBINATION_PRODUCT

L606 inhalation suspension

L606

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females ≥18 and ≤80 years of age.
• Diagnosed with
• PAH belonging to at least 1 of the following subgroups of Group 1 pulmonary hypertension (PH) per European Society of Cardiology/European Respiratory Society Guidelines for the diagnosis and treatment of PH at least 1 year prior to screening. or
• PH-ILD Group 3 European Society of Cardiology/European Respiratory Society Guidelines for the diagnosis and treatment of PH. Subjects with Group 3 PH that is not related to underlying ILD are not eligible.
• Subjects with PAH: Documentation of having PAH as confirmed by RHC meeting the following criteria:
• i. Mean PAP \>20 mmHg. ii. Pulmonary arterial wedge pressure ≤15 mmHg. iii. Pulmonary vascular resistance \>3 Wood units. Subjects with PH-ILD: Confirmation of the underlying ILD must be based on HRCT imaging with demonstration of diffuse parenchymal lung disease and documented by the Investigator or radiology report. Subjects may have any form of ILD or CPFE.
• NYHA functional class II, III, or IV at the screening visit.
• Can complete a screening 6MWD of ≥150 meters
• For subjects with PAH: \>65% of predicted and FEV1/FVC ratio \>65% at screening. For subjects with PH-ILD: \>40% of predicted and FEV1/FVC ratio \>70% at screening.

You may not qualify if:

• LVEF of ≤45% on a historical echocardiogram.
• History of sleep apnea, or left-sided heart disease (including but not limited to aortic or mitral valve disease, pericardial constriction, restrictive or congestive cardiomyopathy, or coronary artery disease) per investigator's discretion.
• Experienced an acute exacerbation of disease or hospitalization for any reason within 30 days of signing the ICF or prior to baseline.
• Musculoskeletal disorder (eg, arthritis affecting the lower limbs, recent hip or knee joint replacement) or any disease that would likely be the primary limit to ambulation or subject is connected to a machine that is not portable enough to allow for a 6MWT.

Sponsors & Collaborators

• Liquidia Technologies, Inc.: lead
• PPD Development, LP: collaborator

Study Sites (8)

Arizona Pulmonary Specialists

Scottsdale, Arizona, 85258, United States

Location

VA Greater Los Angeles Healthcare

Los Angeles, California, 90073, United States

Location

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

Location

University of South Florida

Tampa, Florida, 33606, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029-6504, United States

Location

University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States

Location

Summit Health Eastside Clinic

Bend, Oregon, 97701, United States

Location

Baylor Scott and White Research Institute

Temple, Texas, 76508, United States

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Respiratory Aspiration

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Cohort A: Tyvaso stabilized PAH or PH-ILD patients Cohort B: Prostacyclin naive - PAH patients

Study Record Dates

• First Submitted: December 21, 2020
• First Posted: December 31, 2020
• Study Start: August 1, 2021
• Primary Completion: March 20, 2025
• Study Completion (Estimated): March 31, 2031
• Last Updated: April 1, 2026

Record last verified: 2026-03

Locations

US (8)