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Pulmonary HypertensionPhase 2NCT03727451

A Dose Escalation Study to Assess the Safety and Efficacy of Pulsed Inhaled Nitric Oxide in Subjects With Pulmonary Fibrosis or Sarcoidosis

A Phase 2 study testing iNO in people with Pulmonary Hypertension and Pulmonary Fibrosis, run by Bellerophon at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

A phase 2b, open label study to assess the safety and efficacy of increasing doses of pulsed, inhaled nitric oxide (iNO) in subjects with pulmonary fibrosis and sarcoidosis on long term oxygen therapy followed by a long term extension study Read more about this study

Who is it for?
Ages 18 to 85 years
What is being tested?
iNO
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
6 sitesUnited States
Study dates
30 Jan 2019to 15 Jun 2022
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Signed informed consent
  • A primary diagnosis of sarcoidosis as defined by the ATS/ERS/WASOG statement or pulmonary fibrosis associated with one of the following conditions:
  • 2.1 Major IIPs (idiopathic interstitial pneumonias) diagnosed or suspected as one of the following:
  • Idiopathic pulmonary fibrosis
  • Idiopathic nonspecific interstitial pneumonia
  • Respiratory bronchiolitis-interstitial lung disease
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  • 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
  • 2.2 Chronic hypersensitivity pneumonitis
  • 2.3 Occupational lung disease
  • 2.4 Connective tissue disease with evidence of significant pulmonary fibrosis
  • Intermediate or high probability of PH by echocardiogram as assessed by local Radiologist/Investigator, or PH as determined by a right heart catheterization (RHC) within 5 years prior to Baseline with the following parameters:
  • Pulmonary vascular resistance (PVR) ˃3 Wood Units (WU) (320 dynes.sec.cm-5)
  • A left ventricular end diastolic pressure (LVEDP) or pulmonary capillary wedge pressure (PCWP) ≤ 15 mmHg
  • A mean pulmonary arterial pressure (mPAP) of ≥ 25 mmHg
  • 6MWD ≥ 100 meters and ≤ 450 meters
  • WHO Functional Class II-IV
  • Forced Vital Capacity ≥ 40% predicted within last 6 weeks prior to screening
  • Females of childbearing potential must have a negative pre-treatment pregnancy test (urine).
  • Age between 18 and 85 years (inclusive)
  • Clinically stable for at least 4 weeks prior to Baseline in the opinion of the Investigator
  • If on therapy for their parenchymal lung disease and/or sarcoidosis, then the subject should be on a stable well-tolerated dose of the medication(s) for at least 4 weeks prior to enrollment.

Who cannot join

  • Use of any type of PAH specific therapies
  • Episodes of disease worsening within 3 months prior to Baseline
  • Pregnant or breastfeeding females at Screening
  • Administered L-arginine within 1 month prior to Screening
  • Any subject who has been enrolled in any previous clinical study with inhaled NO administered through pulsed delivery
  • On more than 6 L/min of oxygen at rest by nasal cannula for less than 4 weeks
Show all 14Show fewer
  • Evidence of any connective tissue disease with FVC > 60% in the last 6 months prior to screening unless there is evidence of moderate to severe fibrosis on CT scan in the opinion of the local radiologist/Investigator
  • Evidence of clinically significant combined pulmonary fibrosis with emphysema (CPFE) if > 15% of lung fields by CT scan show evidence of emphysema in the opinion of the local radiologist/Investigator
  • For subjects with sarcoidosis, clinically significant evidence of pulmonary fibrosis on CT scan in the opinion of the local radiologist/Investigator and FVC ≥80% predicted
  • For subjects continuing on open label therapy, the concurrent use of the INOpulse device with a CPAP/BiPAP, or any other positive pressure device
  • Significant heart failure in the opinion of the Investigator
  • LVEF<40% or
  • PCWP on last RHC>15 mmHg (unless concurrent LVEDP <15 mmHg) or
  • Significant diastolic dysfunction on echocardiogram

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • University of Cincinnati

    Cincinnati, Ohio, 45219

  • Inova Heart and Lung Vascular Institute

    Falls Church, Virginia, 22042

  • University of Miami

    Miami, Florida, 33125

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Temple University

    Philadelphia, Pennsylvania, 19140

  • University of Washington Medical Center

    Seattle, Washington, 98195

Other trials in United States

About this study

Official title: A Dose Escalation Study to Assess the Safety and Efficacy of Pulsed, Inhaled Nitric Oxide in Subjects With Pulmonary Hypertension Associated With Pulmonary Fibrosis or Sarcoidosis on Long Term Oxygen Therapy Followed by an Optional Open-Label Long Term Extension Safety Study

A phase 2b, open label study to assess the safety and efficacy of increasing doses of pulsed, inhaled nitric oxide (iNO) in subjects with pulmonary fibrosis and sarcoidosis on long term oxygen therapy followed by a long term extension study

Read the detailed description

A phase 2b, open label study to assess the hemodynamic effects of increasing doses of pulsed, inhaled nitric oxide (iNO) in subjects with pulmonary hypertension associated with pulmonary fibrosis or sarcoidosis on long term oxygen therapy followed by an open label extension study

Being tested
iNO
Keywords
Pulmonary Hypertension, Pulmonary Fibrosis, Sarcoidosis
Medical subject headings
Hypertension, Pulmonary, Pulmonary Fibrosis, Sarcoidosis, Pulmonary, Sarcoidosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • iNOCombination product

    Also called iNOPulse

    inhaled nitric oxide

Study groups in NCT03727451
Study groupRoleWhat they receive
PH-Pulmonary FibrosisExperimentalPart 1: 1st 4 subjects will be treated with iNO 30, 45, 75 mcg/kg IBW/hr 2nd 4 subjects will be treated with iNO 45, 75, 125 mcg/kg IBW/hr Part 2: Optional open label long term extension at the optimal dose as identified in Part 1
Receives: Combination Product: iNO
PH-SarcoidosisExperimentalPart 1: 1st 4 subjects will be treated with iNO 30, 45, 75 mcg/kg IBW/hr 2nd 4 subjects will be treated with iNO 45, 75, 125 mcg/kg IBW/hr Part 2: Optional Open label long term extension at the optimal dose as identified in Part 1
Receives: Combination Product: iNO

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Measurement of mean PAP

    Mean pulmonary arterial pressure (mPAP) will be measured at iNO 30, 45, 75 and 125 mcg/kg IBW/hr

    Measured: During a single right heart catheterization procedure

  2. Measurement of PCWP

    Pulmonary capillary wedge pressure (PCWP) will be measured at iNO 30, 45, 75 and 125 mcg/kg IBW/hr

    Measured: During a single right heart catheterization procedure

  3. Measurement of PVR

    Pulmonary vascular resistance (PVR) will be measured at iNO 30, 45, 75 and 125 mcg/kg IBW/hr

    Measured: During a single right heart catheterization procedure

  4. Measurement of CO

    Cardiac output (CO) will be measured at iNO 30, 45, 75 and 125 mcg/kg IBW/hr

    Measured: During a single right heart catheterization procedure

  5. Change in 6MWD from Baseline to 16 Weeks

    Change in 6 minute walk distance

    Measured: 16 weeks

Secondary outcomes (7)

  1. Incidence and Severity of Treatment Emergent Adverse Events

    Including adverse events related to device deficiency

    Measured: During a single right heart catheterization procedure

  2. Pulmonary Rebound

    Symptoms associated with acute withdrawal of iNO: systemic arterial oxygen desaturation, hypoxemia, bradycardia, tachycardia, systemic hypotension, shortness of breath, near-syncope and syncope

    Measured: During a single right heart catheterization procedure

  3. Distance Saturation Product (DSP)

    Difference in DSP from baseline to Week 16

    Difference in DSP from baseline to 16 weeks

    Measured: 16 weeks

  4. Dyspnea

    Difference in dyspnea as measured by UCSD Medical Center Pulmonary Rehabilitation Program Shortness of Breath Questionnaire on a scale from 0 (none at all) to 5 (maximal or unable to do because of breathlessness) from baseline to Week 16

    Measured: 16 weeks

  5. Quality of Life Assessment

    Difference in disease specific Quality of Life as measured by St. George's Respiratory Questionnaire

    Measured: 16 weeks

  6. Incidence of Adverse Events and Serious Averse Events

    Evaluation of adverse events and serious adverse events

    Measured: Through study completion; an average of 1 year

  7. Integral Distance Saturation Product (IDSP)

    Difference in IDSP from baseline to Week 16

    Measured: 16 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Nov 2018
  2. Study start30 Jan 2019
  3. Primary completion7 Sep 2021
  4. Study completion15 Jun 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
17 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03727451
Sponsor's protocol ID
PULSE-PHPF-002
Status last verified
Feb 2022
First submitted
30 Oct 2018
Last updated
10 Aug 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing iNO in people with Pulmonary Hypertension and Pulmonary Fibrosis, run by Bellerophon at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 27 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Measurement of mean PAP (time frame: During a single right heart catheterization procedure). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03727451.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2022.

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