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

Last updated: October 8, 2024
Sponsor: Liquidia Technologies, Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

Vascular Diseases

Williams Syndrome

Pulmonary Arterial Hypertension

Treatment

L606 inhalation suspension

Clinical Study ID

NCT04691154
PBI L606p3
  • Ages 18-80
  • All Genders

Study 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; also evaluate the steady-state pharmacokinetics (PK) of L606 as compared to Tyvaso, effects on exercise ability, quality of life, and treatment satisfaction with L606.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  1. Able to understand and complete study requirements and provide written informedconsent.

  2. Males and females ≥18 and ≤80 years of age at the time of informed consent. Allsexually active male subjects and female subjects of childbearing potential must usean acceptable, highly effective method of contraception.

  3. Diagnosed with

  4. PAH belonging to at least 1 of the following subgroups of Group 1 pulmonaryhypertension (PH) per European Society of Cardiology/European RespiratorySociety Guidelines for the diagnosis and treatment of PH at least 1 year priorto screening. or

  5. PH-ILD Group 3 European Society of Cardiology/European Respiratory SocietyGuidelines for the diagnosis and treatment of PH. Subjects with Group 3 PH thatis not related to underlying ILD are not eligible.

  6. Subjects with PAH: Documentation of having PAH as confirmed by RHC within 12 monthsprior to screening and meeting the following criteria: i. Mean PAP >20 mmHg. ii. Pulmonary arterial wedge pressure ≤15 mmHg. iii. Pulmonaryvascular resistance >3 Wood units. Subjects with PH-ILD: Confirmation of theunderlying ILD must be based on HRCT imaging conducted within the past 12 monthsprior to randomization with demonstration of diffuse parenchymal lung disease anddocumented by the Investigator or radiology report. Subjects may have any form ofILD or CPFE.

  7. NYHA functional class II, III, or IV at the screening visit.

  8. Can complete a screening 6MWD of ≥150 meters

  9. For subjects with PAH: >65% of predicted and FEV1/FVC ratio >65% at screening. Forsubjects with PH-ILD: >40% of predicted and FEV1/FVC ratio >70% at screening.

Exclusion

Key Exclusion Criteria:

  1. LVEF of ≤45% on a historical echocardiogram within 6 months of screening.

  2. History of sleep apnea, or left-sided heart disease (including but not limited toaortic or mitral valve disease, pericardial constriction, restrictive or congestivecardiomyopathy, or coronary artery disease) per investigator's discretion.

  3. Experienced an acute exacerbation of disease or hospitalization for any reasonwithin 30 days of signing the ICF or prior to baseline.

  4. Systolic blood pressure <90 mmHg or ≥160 mmHg at baseline.

  5. Screening electrocardiogram (ECG) with QTcF >525 ms.

  6. Musculoskeletal disorder (eg, arthritis affecting the lower limbs, recent hip orknee joint replacement) or any disease that would likely be the primary limit toambulation or subject is connected to a machine that is not portable enough to allowfor a 6MWT.

  7. Alanine aminotransferase or aspartate aminotransferase levels >3 × upper limit ofnormal reference range, clinically significant liver disease/dysfunction, or knownChild-Pugh Class C hepatic disease.

  8. Estimated glomerular filtration rate <30 mL/min/1.73 m2 or requires dialysis.

Study Design

Total Participants: 28
Treatment Group(s): 1
Primary Treatment: L606 inhalation suspension
Phase: 3
Study Start date:
August 01, 2021
Estimated Completion Date:
July 31, 2025

Study Description

This Phase 3, 2-part, open-label, multicenter study aims to demonstrate the safety and tolerability of repeated doses of L606 in patients with PAH or PH-ILD. The current Phase 3 study will help determine the short-term and long-term safety and tolerability of L606 in this patient population. The study will also evaluate the steady-state pharmacokinetics (PK) of L606 as compared to Tyvaso, effects on exercise ability (6-minute walk distance [6MWD] and Borg Dyspnea Score), quality of life (QoL), and treatment satisfaction with L606.

Subjects who meet all the inclusion criteria and none of the exclusion criteria will be enrolled to one of the 2 cohorts:

Cohort A: Subjects with PAH or PH-ILD receiving prior stable doses of Tyvaso (4 times daily) and willing to switch to L606 (twice daily).

Cohort B: Subjects with PAH (not initially on prostacyclin therapy) who are likely to receive clinical benefit from inhaled treprostinil based on the opinion of the investigator.

Cohort A subjects will sequentially participate in the MSP for 2 weeks and the OEP for 46 weeks. A subset of up to 15 subjects from Cohort A will also participate in a PK substudy. Cohort B subjects will sequentially participate in the MSP for 12 weeks and the OEP for 36 weeks.

Connect with a study center

  • Arizona Pulmonary Specialists

    Phoenix, Arizona 85012
    United States

    Site Not Available

  • VA Greater Los Angeles Healthcare

    Los Angeles, California 90073
    United States

    Site Not Available

  • Mayo Clinic Jacksonville

    Jacksonville, Florida 32224
    United States

    Site Not Available

  • Brigham and Womens Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • UPMC Montefiore

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

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