Pulmonary Arterial HypertensionPhase 3NCT03602781
Study of PAH Subjects With LTOT Use That Have Demonstrated Improved Exercise Tolerance With the Use of Inhaled Nitric
A Phase 3 study testing iNO in people with Pulmonary Arterial Hypertension, run by Bellerophon Pulse Technologies at 4 sites in 2 countries.
Trial summary
Study of PAH Subjects with LTOT Use that have Demonstrated Improved Exercise Tolerance with the use of Inhaled Nitric Oxide Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- iNO
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Aug 2018to Jun 2020
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed Informed Consent Form prior to the initiation of any study mandated procedures or assessments
- Subjects must be enrolled in the PULSE PAH-004 clinical trial and must have been on LTOT and on open-label treatment with iNO 75 mcg/kg IBW/hr for at least 4 months
- Subjects must have achieved ≥ 30 meter improvement in 6MWD after 4, 8 or 12 months of open-label treatment with iNO 75 mcg/kg IBW/hr as compared to either their PULSE PAH-004 Week 2 end of Run-in OR End of Study (EOS)in PULSE-PAH-004.
- Subjects are willing and considered in the judgement of the Investigator able to use the INOpulse device continuously for up to 24 hours per day
- Female subjects of childbearing potential must have a negative pregnancy test (serum or urine) at randomization. All female subjects must use an effective method of birth control to avoid pregnancy.
Who cannot join
- Subjects with episodes of worsening of PAH in the last 30 days prior to PULSE PAH-007 Baseline/Randomization
- Subjects that experience Pulmonary Rebound in PULSE-PAH-004
- Change in dose or types of PAH specific therapies in the last 30 days prior to Baseline/Randomization
- Subjects who require treatment with riociguat
- Subjects who early discontinued drug/device usage due to withdrawal of consent or an adverse event requiring termination from treatment in PULSE PAH-004
- Women who are pregnant
- The concurrent use of the INOpulse device with a continuous airway pressure (CPAP), Bilevel Positive Airway Pressure (BiPAP, or any other positive pressure device.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States2 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Bluhm Cardiovascular Institute, Clinical Trials Unit
Chicago, Illinois, 60611
Canada2 sites
Peter Lougheed Centre
Calgary, Alberta, T1Y6J4
Toronto General Hospital, University Health Network
Toronto, Ontario, M5G 2C4
About this study
Official title: Phase 3, Multicenter, Randomized, Double-blind, Placebo Controlled Withdrawal Study of Pulmonary Arterial Hypertension(PAH) Subjects With LTOT Use That Have Demonstrated Improved Exercise Tolerance With the Use of Inhaled Nitric Oxide (INO)
Study of PAH Subjects with LTOT Use that have Demonstrated Improved Exercise Tolerance with the use of Inhaled Nitric Oxide
Read the detailed description
Phase 3, Multicenter, Randomized, Double-blind, Placebo Controlled Withdrawal Study of Pulmonary Arterial Hypertension(PAH) Subjects with LTOT Use that have Demonstrated Improved Exercise Tolerance with the use of Inhaled Nitric Oxide (INO)
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Placebo, iNO
- Keywords
- PAH, Inhaled Nitric Oxide, iNO, Long Term Oxygen Therapy, Oxygen therapy
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Also called Placebo 99.999% Nitrogen
Placebo at a dose setting of 75 mcg/kg IBW/hr
iNODrug
Also called Inhaled Nitric Oxide
iNO at a dose setting of 75 mcg/kg IBW/hr
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Placebo 99.999% Nitrogen | Placebo comparator | Randomized Withdrawal Treatment Period Week 1-8:
Placebo at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day
Long Term Open Label Extension Period:
iNO at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day Receives: Drug: Placebo; Drug: iNO |
| Cohort 2: iNO 75 mcg/kg IBW/hr | Active comparator | Randomized Withdrawal Treatment Period Week 1-8:
iNO at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day
Long Term Open Label Extension Period:
iNO at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day Receives: Drug: iNO |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to clinical worsening during iNO withdrawal for up to 8 weeks
A clinical worsening event is defined as:
- Death (all-cause mortality)
- Atrial septostomy
- Hospitalization due to worsening of PAH
- Need to start additional specific PAH treatment
- Decrease of >15% in 6 Minute Walk Distance from randomization into the study
- Worsening of WHO Functional Class (e.g., from Class II to Class III or IV, OR Class III to Class IV)
Measured: 8 weeks
Secondary outcomes (1)
Difference in clinical worsening events that occur during iNO withdrawal for up to 8 weeks between those treated with iNO ≥ 10 months prior to the start of withdrawal of iNO vs. those treated < 10 months prior to initiation of withdrawal to iNO.
A clinical worsening event is defined as:
- Death (all-cause mortality)
- Atrial septostomy
- Hospitalization due to worsening of PAH
- Need to start additional specific PAH treatment
- Decrease of >15% in 6 Minute Walk Distance from randomization into the study
- Worsening of WHO Functional Class (e.g., from Class II to Class III or IV, OR Class III to Class IV)
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2018
- Study startAug 2018 (estimated)
- Primary completionJun 2019 (estimated)
- Study completionJun 2020 (estimated)
Why it stopped: Decision not to proceed with Study
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bellerophon Pulse Technologies
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Responsible party
- Sponsor
- Investigators
- Ashika Ahmed, MD, Study director, Bellerophon Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03602781
- Sponsor's protocol ID
- PULSE-PAH-007
- Status last verified
- Jul 2018
- First submitted
- 6 Jul 2018
- Last updated
- 21 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3, Multicenter, Randomized, Double-blind, Placebo Controlled Withdrawal Study of Pulmonary Arterial Hypertension(PAH) Subjects With LTOT Use That Have Demonstrated Improved Exercise Tolerance With the Use of Inhaled Nitric Oxide (INO)
- ClinicalTrials.gov ID
- NCT03602781
- Acronym / sponsor ID
- PULSE-PAH-007
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- PAH, Inhaled Nitric Oxide, iNO, Long Term Oxygen Therapy, Oxygen therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Decision not to proceed with Study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Bellerophon Pulse TechnologiesIndustry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Study start
- Aug 2018 (estimated)
- Primary completion
- Jun 2019 (estimated)
- Study completion
- Jun 2020 (estimated)
- First posted
- 27 Jul 2018 (actual)
- 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.
Study of PAH Subjects with LTOT Use that have Demonstrated Improved Exercise Tolerance with the use of Inhaled Nitric Oxide
Detailed description
Phase 3, Multicenter, Randomized, Double-blind, Placebo Controlled Withdrawal Study of Pulmonary Arterial Hypertension(PAH) Subjects with LTOT Use that have Demonstrated Improved Exercise Tolerance with the use of Inhaled Nitric Oxide (INO)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Placebo 99.999% Nitrogen | Placebo comparator | Drug: Placebo, Drug: iNORandomized Withdrawal Treatment Period Week 1-8: Placebo at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day Long Term Open Label Extension Period: iNO at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day |
| Cohort 2: iNO 75 mcg/kg IBW/hr | Active comparator | Drug: iNORandomized Withdrawal Treatment Period Week 1-8: iNO at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day Long Term Open Label Extension Period: iNO at a dose setting of 75 mcg/kg IBW/hr for up to 24 hr/day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Signed Informed Consent Form prior to the initiation of any study mandated procedures or assessments
- Subjects must be enrolled in the PULSE PAH-004 clinical trial and must have been on LTOT and on open-label treatment with iNO 75 mcg/kg IBW/hr for at least 4 months
- Subjects must have achieved ≥ 30 meter improvement in 6MWD after 4, 8 or 12 months of open-label treatment with iNO 75 mcg/kg IBW/hr as compared to either their PULSE PAH-004 Week 2 end of Run-in OR End of Study (EOS)in PULSE-PAH-004.
- Subjects are willing and considered in the judgement of the Investigator able to use the INOpulse device continuously for up to 24 hours per day
- Female subjects of childbearing potential must have a negative pregnancy test (serum or urine) at randomization. All female subjects must use an effective method of birth control to avoid pregnancy.
Key exclusion criteria (7 listed)
- Subjects with episodes of worsening of PAH in the last 30 days prior to PULSE PAH-007 Baseline/Randomization
- Subjects that experience Pulmonary Rebound in PULSE-PAH-004
- Change in dose or types of PAH specific therapies in the last 30 days prior to Baseline/Randomization
- Subjects who require treatment with riociguat
- Subjects who early discontinued drug/device usage due to withdrawal of consent or an adverse event requiring termination from treatment in PULSE PAH-004
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 |
|---|---|---|
| PlaceboAlso: Placebo 99.999% NitrogenPlacebo at a dose setting of 75 mcg/kg IBW/hr | Drug | Cohort 1: Placebo 99.999% Nitrogen |
| iNOAlso: Inhaled Nitric OxideiNO at a dose setting of 75 mcg/kg IBW/hr | Drug | Cohort 1: Placebo 99.999% Nitrogen, Cohort 2: iNO 75 mcg/kg IBW/hr |
Primary outcome
Time to clinical worsening during iNO withdrawal for up to 8 weeks
Time frame: 8 weeks
A clinical worsening event is defined as: 1. Death (all-cause mortality) 2. Atrial septostomy 3. Hospitalization due to worsening of PAH 4. Need to start additional specific PAH treatment 5. Decrease of >15% in 6 Minute Walk Distance from randomization into the study 6. Worsening of WHO Functional Class (e.g., from Class II to Class III or IV, OR Class III to Class IV)
1 secondary outcome
Difference in clinical worsening events that occur during iNO withdrawal for up to 8 weeks between those treated with iNO ≥ 10 months prior to the start of withdrawal of iNO vs. those treated < 10 months prior to initiation of withdrawal to iNO.
Time frame: 8 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2023.
- Individual participant dataSharing planned: No
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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.
Ashika Ahmed, MD study director
Bellerophon Therapeutics
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing iNO in people with Pulmonary Arterial Hypertension, run by Bellerophon Pulse Technologies at 4 sites in 2 countries.
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 5 inclusion and 7 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: withdrawn, as of the sponsor's last update on 21 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Canada. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was withdrawn before enrolling participants, so there are no results.
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?
Time to clinical worsening during iNO withdrawal for up to 8 weeks (time frame: 8 weeks). 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 21 Feb 2023.