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Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects with PPF

UCI Specialty Area: Pulmonology
Principal Investigator: Bassam Yaghmour
Official Title: A Randomized, Double-blind, Placebo-controlled, Multinational, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects with Progressive Pulmonary Fibrosis (TETON-PPF)
A Study On:
Lung - Pulmonary

Study Description

Study RIN-PF-305 is designed to evaluate the safety and efficacy of inhaled treprostinil in subjects with progressive pulmonary fibrosis (PPF) over a 52-week period.

Study RIN-PF-305 is a Phase 3, multinational, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of inhaled treprostinil in subjects with PPF over a 52-week period. Subjects will be randomly allocated 1:1 to receive inhaled treprostinil or placebo. All subjects will initiate inhaled treprostinil or placebo at a dose of 3 breaths administered 4 times daily (QID) and will titrate to a target dosing regimen of 12 breaths QID. Study drug doses may be titrated up as tolerated, until the target dose or maximum clinically tolerated dose is achieved. Once eligible, 6 Treatment Period visits to the clinic will be required at Weeks 4, 8, 16, 28, 40, and 52.

Efficacy assessments include spirometry (forced vital capacity [FVC]), time to clinical worsening, time to first acute exacerbation of interstitial lung disease (ILD), overall survival, King's Brief Interstitial Lung Disease (K-BILD) questionnaire, plasma N-terminal pro-brain natriuretic peptide (NT-proBNP) concentration, supplemental oxygen use, and lung diffusion capacity (DLCO). Safety assessments include the development of adverse events (AEs)/serious adverse events (SAEs), vital signs, clinical laboratory parameters, and electrocardiogram (ECG) parameters.

Subjects who complete the Week 52 Visit may be offered the opportunity to enter an open-label extension (OLE) study after completing the final study visit.

Eligibility

  1. Subject gives voluntary informed consent to participate in the study.
  2. Subject is greater than or equal to 18 years of age, inclusive, at the time of signing informed consent.
  3. Subject has radiological evidence of pulmonary fibrosis of >10% extent on an HRCT scan in the previous 12 months (confirmed by central review).
  4. Subject has a diagnosis of PPF (other than IPF) that fulfills at least 1 of the following criteria for progression within 24 months of screening despite standard treatment of ILD, as assessed by the Investigator:
  • Clinically significant decline in % predicted FVC based on greater than or equal to 10% relative decline.
  • Marginal decline in % predicted FVC based on greater than or equal to 5% to <10% relative decline combined with worsening of respiratory symptoms.
  • Marginal decline in % predicted FVC based on greater than or equal to 5% to <10% relative decline combined with increasing extent of fibrotic changes on chest imaging.
  • Worsening of respiratory symptoms as well as increasing extent of fibrotic changes on chest imaging
  1. FVC greater than or equal to 45% predicted at Screening (confirmed by central review).
  2. Subjects must be on 1 of the following:
  • On nintedanib or pirfenidone for greater than or equal to 90 days prior to Baseline and in the Investigator's opinion, are planning to continue treatment through the study
  • Not on treatment with nintedanib or pirfenidone for greater than or equal to 90 days prior to Baseline and in the Investigator's opinion, not planning to initiate either treatment during the study.

Concomitant use of both nintedanib and pirfenidone is not permitted.

  1. Subjects treated with immunosuppressive agents (eg, mycophenolate, methotrexate, azathioprine, oral corticosteroids, rituximab) need to be on treatment for at least 120 days prior to Baseline and, in the Investigator's clinical opinion, must be refractory to treatment.
  2. Women of childbearing potential must be non-pregnant (as confirmed by a urine pregnancy test at Screening and Baseline) and non-lactating, and will agree to do 1 of the following:
  • Abstain from intercourse (when it is in line with their preferred and usual lifestyle)
  • Use 2 medically acceptable, highly effective forms of contraception for the duration of the study, and at least 30 days after discontinuing study drug.
  1. Males with a partner of childbearing potential must agree to use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug.
  2. In the opinion of the Investigator, the subject is able to communicate effectively with study personnel, and is considered reliable, willing, and likely to be cooperative with protocol requirements, including attending all study visits.

Other protocol-defined Inclusion/

Exclusion Criteria apply

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University of California, Irvine - Study Information Sheet

UCI Center for Clinical Research Potential Participant Database

Lead Researcher
Maheswari Senthil, MD
UCI Center for Clinical Research

In the instance of parental permission, “You” refers to “Your child.”

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