Clinical trial NCT05747508: A Study to Assess Pulsed Inhaled Nitric Oxide in Subjects With Pulmonary Hypertension Associated With Pulmonary Fibrosis
Official title: A Randomized, Double-Blind, Placebo-Controlled Dose Escalation Clinical Study to Assess the Safety and Efficacy of Pulsed Inhaled Nitric Oxide (iNO) in Subjects With Pulmonary Hypertension Associated With Pulmonary Fibrosis on Long Term Oxygen Therapy (Part 1 and Part 2)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Pulmonary Fibrosis
Pulmonary Hypertension
Keywords: Pulmonary Fibrosis (PF)
Pulmonary Hypertension
Inhaled Nitric Oxide
iNO
Long Term Oxygen Therapy
Oxygen
Lead sponsor: Bellerophon
Sponsor class: INDUSTRY
First posted: 2023-02-28
Enrollment: 85
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A randomized, double-blind, placebo-controlled dose escalation study to assess the safety and efficacy of pulsed, inhaled nitric oxide (iNO) in subjects with pulmonary fibrosis on long term oxygen therapy.

Detailed description: A Phase 2b, randomized, double-blind, placebo-controlled dose escalation clinical study to assess the safety and efficacy of pulsed, inhaled nitric oxide in subjects with and without pulmonary hypertension associated with pulmonary fibrosis on long term oxygen therapy (Part 1 and Part 2)

Eligibility criteria: Inclusion Criteria:

* Diagnosed with pulmonary fibrosis by high resolution CT scan performed in the 6 months prior to screening associated with one of the following conditions and confirmed using one of the following guidelines, as per American Thoracic Society (AGS) / European Respiratory Society (ERS) / Japanese Respiratory Society (JRS) / Latin American Thoracic Association (ALAT):

  * Major idiopathic interstitial pneumonias (IIPs) diagnosis or suspected as one of the following:

    * Idiopathic pulmonary fibrosis
    * Idiopathic nonspecific interstitial pneumonia
    * Respiratory bronchiolitis-interstitial lung disease
    * Desquamative interstitial pneumonia
    * Cryptogenic organizing pneumonia
    * Acute interstitial pneumonia
  * Rare IIPs diagnosis by one of the following:

    * Idiopathic lymphoid interstitial pneumonia
    * Idiopathic pleuroparenchymal fibroelastosis
  * Unclassifiable idiopathic interstitial pneumonias
  * Chronic hypersensitivity pneumonitis
  * Occupational lung disease
* Have been using oxygen therapy by nasal cannula for at least 4 weeks
* 6-Minute Walk Distance (6MWD) ≥ 100 meters and ≤ 450 meters at screening and Baseline/Randomization visits
* World Health Organization (WHO) Functional Class II-IV
* Forced Vital Capacity ≥ 40% predicated within the last 6 months prior to the screening run-in period
* Age between 18 and 85 years (inclusive)

Exclusion Criteria:

* Pregnant or breastfeeding females at Screening
* In the last 6 months prior to screening, evidence of any connective tissue disease with FVC \> 60% unless there is evidence of moderate to severe fibrosis on CT scan in the opinion of the local radiologist/Investigator

Primary outcomes:
measure: Efficacy of escalating doses of iNO in patient's life participation as assessed in minutes per day of moderate to vigorous activity.
timeFrame: Change from baseline to week 8 or 16
Secondary outcomes:
Other outcomes:
measure: Change in 6-minute walking test (6MWT) from baseline
timeFrame: Change from baseline to week 8 or 16
measure: Difference in activity as measured using ActiGraph devices
timeFrame: Change from baseline to week 8 or 16
measure: Percentage of patients with ≥15% decrease in activity
timeFrame: Change from baseline to to week 8 or 16
measure: Difference in dyspnea as measured by University of California San Diego Shortness of Breath Questionnaire
timeFrame: Change from baseline to to week 8 or 16
measure: Difference in St. George Respiratory Questionnaire and sub-groups
timeFrame: Change from baseline to to week 8 or 16
measure: Percentage of patients with a clinically meaningful difference (reduction of at least 4 points) in the disease-specific quality of life measured using the disease specific St. George Respiratory Questionnaire (SGRQ)
timeFrame: Change from baseline to to week 8 or 16
measure: Change in N-terminal (NT)-ProBNP (absolute and percentage)
timeFrame: Change from baseline to week 8 or 16
measure: Time to clinical improvement
timeFrame: Baseline to week 8 or 16
measure: Time to clinical worsening
timeFrame: Baseline to week 8 or 16
measure: Incidence and severity of treatment emergent adverse events, including those related to INOpulse® device deficiency
timeFrame: Baseline to week 8 or 16
measure: Adverse events that may be due to rebound associated with a temporal acute withdrawal of investigational study drug
timeFrame: Baseline to week 8 or 16
measure: Change in diffusing capacity of the lungs for carbon monoxide (DLCO)
timeFrame: Baseline to week 8 or 16
measure: Mortality
timeFrame: Baseline to week 8 or 16
measure: Hospitalization (all cause and for cardiopulmonary or other cause)
timeFrame: Baseline to week 8 or 16
measure: Change in Forced Expiratory Volume at 1 minute (FEV1)
timeFrame: Baseline to week 8 or 16
measure: Change in Forced Vital Capacity (FVC)
timeFrame: Baseline to week 8 or 16
Locations:
city: Phoenix
country: United States
facility: Arizona Pulmonary Specialists
state: Arizona
city: Los Angeles
country: United States
facility: University of California
state: California
city: Sacramento
country: United States
facility: University of California Davis Health
state: California
city: Aurora
country: United States
facility: University of Colorado Hospital
state: Colorado
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Pensacola
country: United States
facility: Avanza Medical Research Center
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Austell
country: United States
facility: Piedmont Healthcare Pulmonary & Critical Care Research
state: Georgia
city: Chicago
country: United States
facility: Loyola University
state: Illinois
city: Louisville
country: United States
facility: Norton Pulmonary Specialists
state: Kentucky
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Chesterfield
country: United States
facility: The Lung Research Center (St. Luke's)
state: Missouri
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati College of Medicine
state: Ohio
city: Philadelphia
country: United States
facility: Thomas Jefferson University Korman Respiratory Institute
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University Hospital
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Sciences
state: Utah
city: Falls Church
country: United States
facility: Inova Heart and Vascular Institute Advanced Lung Disease Clinic
state: Virginia
city: Richmond
country: United States
facility: Pulmonary Associates of Richmond
state: Virginia
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
Study officials:
affiliation: Bellerophon Therapeutics
name: Ashika Ahmed, MD
role: STUDY_DIRECTOR
References: