Pulmonary Arterial HypertensionPhase 2PHianoNCT01265888
Study in Subjects With PAH and PH Secondary to IPF Using Inhaled GeNOsyl.
A Phase 2 study testing Inhaled Nitric Oxide in people with Pulmonary Arterial Hypertension and Idiopathic Pulmonary Fibrosis, run by Geno LLC at 10 sites in United States.
Trial summary
A Phase 2 open label, dose escalation study to find the minimally and maximum effective dose (dose beyond which no further effect on PVR is seen) of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pulmonary Arterial Hypertension, Idiopathic Pulmonary Fibrosis
- What is being tested?
- Inhaled Nitric Oxide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 10 sitesUnited States
- Study dates
- Mar 2011to Sep 2016
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- PAH and PH-IPF
- WHO Functional Class (or equivalent classification) II - IV.
- Subjects using supplemental oxygen must be receiving a stable course of therapy for a minimum of 14 days prior to study drug administration.
- All subjects' oxygen saturation must be > or = to 88% at time of treatment
- Echocardiogram within 6 months of baseline showing no signs of clinically significant left sided heart disease
- Females of child-bearing potential with a negative urine pregnancy test, or a documented surgical sterilization, or is post-menopausal prior to administration of investigational product. Females of childbearing potential must be practicing adequate birth control.
Show all 20Show fewer
- PAH (WHO Group 1) ONLY-Inclusion
- Documented diagnosis of WHO Group 1 PAH, (limited to, idiopathic, heritable, drug and toxin induced, associated with connective tissue disease, portal hypertension, repaired congenital heart disease, HIV); documented by a previous RHC and hemodynamics consistent with PAH, WHO Group 1
- Pulmonary Function Testing within 6 months prior to screening/enrollment shows no evidence of interstitial lung disease (TLC<70%) or obstructive lung disease (FEV1/FVC ratio <50%)
- Receiving a stable course of approved PAH oral mono therapies for a minimum of 14 days prior to treatment period
- Must be 18-80 year of age
- PH-IPF (WHO Group 3) ONLY-Inclusion
- Documented diagnosis of probable or definite IPF using ATS/ERS criteria
- Previous transbronchial biopsy, if performed, shows no features to support a definitive alternative diagnosis
- Previous bronchoalveolar lavage, if performed, shows no features that provides an alternative diagnosis
- FVC > or = 40% within 6 months of baseline visit
- Diagnosis of PH based on hemodynamic requirements
- Age 40-85.
- Diagnosis of IPF > or = 3 months prior to study drug administration
- Diagnosis of PH based on the following hemodynamic criteria (PAPm > or = 25 mmHg (at rest) / PCWP or LVED < or =15 mmHg and / PVR >3 Wood Units)
Who cannot join
- PAH and PH-IPF
- Have any other disease associated with pulmonary hypertension (i.e. Group II, IV or V).
- Documented uncontrolled systemic hypertension.
- Left ventricular ejection fraction (LVEF) < 40%.
- Have chronic kidney disease stage IV or worse, or requires dialysis.
- Be receiving an investigational drug, have in place an investigational device, or have participated in an investigational drug study within past 30 days.
Show all 22Show fewer
- Have had an atrial septostomy.
- Have anemia or any other ongoing condition that would interfere with the interpretation of study assessments.
- Have any serious or life-threatening disease other than conditions associated with PAH or PH-IPF (e.g. malignancy requiring aggressive chemotherapy, renal dialysis, etc.)
- Significant, ongoing alcohol or drug abuse, or unstable psychiatric status.
- Receiving inhaled or parenteral prostacyclins or a non-approved combination of approved oral PAH therapy.
- Pregnant or lactating subjects
- PAH (WHO Group 1) ONLY-Exclusion
- Have had any changes to chronic therapy (including but not limited to oxygen, a different category of vasodilator, a diuretic, digoxin) for PAH added within 14 days of study drug administration. Anticoagulants are allowed to be discontinued per institutional standard of care.
- History of untreated sleep apnea within three months of study drug administration.
- History of hemodynamically significant left-sided heart disease or evidence of left-sided heart disease.
- PH-IPF (WHO Group 3) ONLY-Exclusion
- Diagnosis of PH primarily due to etiology other than IPF.
- FEV/FVC ratio < 60% documented within 6 months of baseline visit.
- Hospitalization for acute exacerbation of IPF within 30 days of baseline visit.
- Recent active pulmonary or upper respiratory tract infection.
- Receiving any approved PAH therapy within 30 days of study drug administration.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
University of Alabama @ Birmingham
Birmingham, Alabama, 35294
Ohio State University, Martha Morehouse Medical Plaza
Columbus, Ohio, 43221
UT Southwestern Medical Center
Dallas, Texas, 75239
National Jewish Health
Denver, Colorado, 80206
VA Greater LA Health Care System-UCLA
Los Angeles, California, 90073
Kentuckiana Pulmonary Associates
Louisville, Kentucky, 40204
University of Medicine and Dentistry of New Jersey
Newark, New Jersey, 07103
University of California- Davis Medical Center
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84132
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Phase 2, Open-Label, Dose-Escalation Study in Subjects With Pulmonary Arterial Hypertension, (PAH, WHO Group 1) and Pulmonary Hypertension Secondary to Idiopathic Pulmonary Fibrosis, (PH-IPF WHO Group 3) Using Inhaled GeNOsyl.
A Phase 2 open label, dose escalation study to find the minimally and maximum effective dose (dose beyond which no further effect on PVR is seen) of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
Read the detailed description
Up to 54 subjects undergoing RHC are planned in this study, and shall include subjects meeting eligibility criteria classified as WHO Group 1 PAH or WHO Group 3 IPF-PH. Subjects will receive inhaled nitric oxide from the GeNOsyl® System to characterize the hemodynamic response and evaluate safety and tolerability.
Dose cohorts of approximately 5, 15, and 20 ppm nitric oxide in air will be studied. Different dose levels will be achieved by varying the flow rate of the drug substance (80 ppm NO2 in balance air) delivered to the subject via nasal cannula. Each subject will receive two different doses of inhaled nitric oxide separated by a placebo (medical grade air or supplemental oxygen) washout. Eligible subjects will be assigned to a dosing cohort in an escalating manner to receive study drug (80 ppm nitric oxide in air.)
- Being tested
- Inhaled Nitric Oxide
- Keywords
- PAH, PH IPF
- Medical subject headings
- Pulmonary Arterial Hypertension, Idiopathic Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inhaled Nitric OxideDrug
Also called GeNOsyl
Inhaled nitric oxide 80 ppm via nasal cannula at 1 LPM for 15 minutes followed by placebo washout of 15 minutes inhalation of medical grade air (or prescribed supplemental oxygen), and then inhaled nitric oxide 80 ppm via nasal cannula at 1 LPM
Inhaled Nitric OxideDrug
Also called GeNOsyl
Inhaled nitric oxide 80 ppm via nasal cannula at 1 LPM for 15 minutes followed by placebo washout of 15 minutes inhalation of medical grade air (or prescribed supplemental oxygen), and then inhaled nitric oxide 80 ppm via nasal cannula at 2 LPM
Inhaled Nitric OxideDrug
Also called GeNOsyl
Inhaled nitric oxide 80 ppm via nasal cannula at 1 LPM for 15 minutes followed by placebo washout of 15 minutes inhalation of medical grade air (or prescribed supplemental oxygen), and then inhaled nitric oxide 80 ppm via nasal cannula at 4 LPM
| Study group | Role | What they receive |
|---|---|---|
| Dosing Group 1 | Experimental | 1 Liter per Minute (LPM)of inhaled nitric oxide via nasal cannula: approximately 5 parts per million (ppm) Receives: Drug: Inhaled Nitric Oxide |
| Dosing Group 2 | Experimental | 2 LPM of inhaled nitric oxide via nasal cannula: approximately 15 ppm Receives: Drug: Inhaled Nitric Oxide |
| Dosing Group 3 | Experimental | 4 LPM of inhaled nitric oxide via nasal cannula: approximately 20 ppm Receives: Drug: Inhaled Nitric Oxide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify the minimally and maximum effective doses of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
Assess mean change in pulmonary vascular resistance (PVR) for study drug dose 2 compared to placebo.
Assess change from pre-dose to end-of-hemodynamic assessment for study drug dose 1.
Assess change from placebo to end-of-hemodynamic assessment for study drug dose 2.
Measured: through end of Right Heart Catheterization procedure (Treatment Phase approximately 3 hours)
Secondary outcomes (2)
Assess the safety and tolerability of nitric oxide generated by the GeNOsyl® System in subjects with WHO Group 1 PAH and WHO Group 3 PH-IPF.
Measured: through end of study (approximately 30 days after treatment visit)
Evaluate the pharmacokinetics of total nitrates/nitrites and methemoglobin produced following inhalation of nitric oxide via the GeNOsyl® System.
Individual pharmacokinetic parameters for total nitrates/nitrites and methemoglobin will be summarized with descriptive characteristics.
Measured: up through 24 hrs after treatment period for subjects participating in PK sub-study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2010 (estimated)
- Study startMar 2011
- Primary completionSep 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Single group
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Geno LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gregory Suplick, Study director, Geno LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01265888
- Sponsor's protocol ID
- Protocol # GeNO-P-2010-002
- Other IDs
- PHiano
- Acronym
- PHiano
- Status last verified
- Sep 2016
- First submitted
- 20 Dec 2010
- Last updated
- 19 Sep 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Dose-Escalation Study in Subjects With Pulmonary Arterial Hypertension, (PAH, WHO Group 1) and Pulmonary Hypertension Secondary to Idiopathic Pulmonary Fibrosis, (PH-IPF WHO Group 3) Using Inhaled GeNOsyl.
- ClinicalTrials.gov ID
- NCT01265888
- Acronym / sponsor ID
- PHiano / Protocol # GeNO-P-2010-002
- Keywords
- PAH, PH IPF
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Geno LLCIndustry (pharma, biotech, devices)
- Study start
- Mar 2011
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 23 Dec 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 2 open label, dose escalation study to find the minimally and maximum effective dose (dose beyond which no further effect on PVR is seen) of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
Detailed description
Up to 54 subjects undergoing RHC are planned in this study, and shall include subjects meeting eligibility criteria classified as WHO Group 1 PAH or WHO Group 3 IPF-PH. Subjects will receive inhaled nitric oxide from the GeNOsyl® System to characterize the hemodynamic response and evaluate safety and tolerability.
Dose cohorts of approximately 5, 15, and 20 ppm nitric oxide in air will be studied. Different dose levels will be achieved by varying the flow rate of the drug substance (80 ppm NO2 in balance air) delivered to the subject via nasal cannula. Each subject will receive two different doses of inhaled nitric oxide separated by a placebo (medical grade air or supplemental oxygen) washout. Eligible subjects will be assigned to a dosing cohort in an escalating manner to receive study drug (80 ppm nitric oxide in air.)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dosing Group 1 | Experimental | Drug: Inhaled Nitric Oxide1 Liter per Minute (LPM)of inhaled nitric oxide via nasal cannula: approximately 5 parts per million (ppm) |
| Dosing Group 2 | Experimental | Drug: Inhaled Nitric Oxide2 LPM of inhaled nitric oxide via nasal cannula: approximately 15 ppm |
| Dosing Group 3 | Experimental | Drug: Inhaled Nitric Oxide4 LPM of inhaled nitric oxide via nasal cannula: approximately 20 ppm |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- PAH and PH-IPF
- WHO Functional Class (or equivalent classification) II - IV.
- Subjects using supplemental oxygen must be receiving a stable course of therapy for a minimum of 14 days prior to study drug administration.
- All subjects' oxygen saturation must be > or = to 88% at time of treatment
- Echocardiogram within 6 months of baseline showing no signs of clinically significant left sided heart disease
Key exclusion criteria (22 listed)
- PAH and PH-IPF
- Have any other disease associated with pulmonary hypertension (i.e. Group II, IV or V).
- Documented uncontrolled systemic hypertension.
- Left ventricular ejection fraction (LVEF) < 40%.
- Have chronic kidney disease stage IV or worse, or requires dialysis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Inhaled Nitric OxideAlso: GeNOsylInhaled nitric oxide 80 ppm via nasal cannula at 1 LPM for 15 minutes followed by placebo washout of 15 minutes inhalation of medical grade air (or prescribed supplemental oxygen), and then inhaled nitric oxide 80 ppm via nasal cannula at 1 LPM | Drug | Dosing Group 1 |
| Inhaled Nitric OxideAlso: GeNOsylInhaled nitric oxide 80 ppm via nasal cannula at 1 LPM for 15 minutes followed by placebo washout of 15 minutes inhalation of medical grade air (or prescribed supplemental oxygen), and then inhaled nitric oxide 80 ppm via nasal cannula at 2 LPM | Drug | Dosing Group 2 |
| Inhaled Nitric OxideAlso: GeNOsylInhaled nitric oxide 80 ppm via nasal cannula at 1 LPM for 15 minutes followed by placebo washout of 15 minutes inhalation of medical grade air (or prescribed supplemental oxygen), and then inhaled nitric oxide 80 ppm via nasal cannula at 4 LPM | Drug | Dosing Group 3 |
Primary outcome
Identify the minimally and maximum effective doses of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
Time frame: through end of Right Heart Catheterization procedure (Treatment Phase approximately 3 hours)
Assess mean change in pulmonary vascular resistance (PVR) for study drug dose 2 compared to placebo. Assess change from pre-dose to end-of-hemodynamic assessment for study drug dose 1. Assess change from placebo to end-of-hemodynamic assessment for study drug dose 2.
2 secondary outcomes
Assess the safety and tolerability of nitric oxide generated by the GeNOsyl® System in subjects with WHO Group 1 PAH and WHO Group 3 PH-IPF.
Time frame: through end of study (approximately 30 days after treatment visit)
Evaluate the pharmacokinetics of total nitrates/nitrites and methemoglobin produced following inhalation of nitric oxide via the GeNOsyl® System.
Time frame: up through 24 hrs after treatment period for subjects participating in PK sub-study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2016.
- Individual participant dataSharing planned: Yes
Related trials for Pulmonary Arterial Hypertension
Not yet recruitingPhase 2/3
An Open-Label Extension Study to Evaluate Inhaled Treprostinil SMI in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Not yet recruitingPhase 2
Study of Treprostinil Administered Via Soft Mist Inhaler (SMI) in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Active, not recruitingPhase 1
A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
Pulmonary Arterial Hypertension
RecruitingObservational study
Natural History Study of Biomarkers in Pulmonary Arterial Hypertension
Pulmonary Disease, Pulmonary Hypertension
Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gregory Suplick study director
Geno LLC
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Inhaled Nitric Oxide in people with Pulmonary Arterial Hypertension and Idiopathic Pulmonary Fibrosis, run by Geno LLC at 10 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 20 inclusion and 22 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 19 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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?
Identify the minimally and maximum effective doses of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo. (time frame: through end of Right Heart Catheterization procedure (Treatment Phase approximately 3 hours)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Sep 2016.