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Study of PAH Subjects With LTOT Use That Have Demonstrated Improved Exercise Tolerance With the Use of Inhaled Nitric

Primary Purpose

Pulmonary Arterial Hypertension

Status
Withdrawn
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Placebo
iNO
Sponsored by
Bellerophon Pulse Technologies
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pulmonary Arterial Hypertension focused on measuring PAH, Inhaled Nitric Oxide, iNO, Long Term Oxygen Therapy, Oxygen therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Signed Informed Consent Form prior to the initiation of any study mandated procedures or assessments
  2. 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
  3. 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.
  4. 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
  5. 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.

Exclusion Criteria:

  1. Subjects with episodes of worsening of PAH in the last 30 days prior to PULSE PAH-007 Baseline/Randomization
  2. Subjects that experience Pulmonary Rebound in PULSE-PAH-004
  3. Change in dose or types of PAH specific therapies in the last 30 days prior to Baseline/Randomization
  4. Subjects who require treatment with riociguat
  5. Subjects who early discontinued drug/device usage due to withdrawal of consent or an adverse event requiring termination from treatment in PULSE PAH-004
  6. Women who are pregnant
  7. The concurrent use of the INOpulse device with a continuous airway pressure (CPAP), Bilevel Positive Airway Pressure (BiPAP, or any other positive pressure device.

Sites / Locations

  • Bluhm Cardiovascular Institute, Clinical Trials Unit
  • Medical University of South Carolina
  • Peter Lougheed Centre
  • Toronto General Hospital, University Health Network

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Active Comparator

Arm Label

Cohort 1: Placebo 99.999% Nitrogen

Cohort 2: iNO 75 mcg/kg IBW/hr

Arm Description

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

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

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

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)

Full Information

First Posted
July 6, 2018
Last Updated
February 17, 2023
Sponsor
Bellerophon Pulse Technologies
Collaborators
Worldwide Clinical Trials
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1. Study Identification

Unique Protocol Identification Number
NCT03602781
Brief Title
Study of PAH Subjects With LTOT Use That Have Demonstrated Improved Exercise Tolerance With the Use of Inhaled Nitric
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 Type
Interventional

2. Study Status

Record Verification Date
July 2018
Overall Recruitment Status
Withdrawn
Why Stopped
Decision not to proceed with Study
Study Start Date
August 2018 (Anticipated)
Primary Completion Date
June 2019 (Anticipated)
Study Completion Date
June 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bellerophon Pulse Technologies
Collaborators
Worldwide Clinical Trials

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Arterial Hypertension
Keywords
PAH, Inhaled Nitric Oxide, iNO, Long Term Oxygen Therapy, Oxygen therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1: Placebo 99.999% Nitrogen
Arm Type
Placebo Comparator
Arm Description
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
Arm Title
Cohort 2: iNO 75 mcg/kg IBW/hr
Arm Type
Active Comparator
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Placebo 99.999% Nitrogen
Intervention Description
Placebo at a dose setting of 75 mcg/kg IBW/hr
Intervention Type
Drug
Intervention Name(s)
iNO
Other Intervention Name(s)
Inhaled Nitric Oxide
Intervention Description
iNO at a dose setting of 75 mcg/kg IBW/hr
Primary Outcome Measure Information:
Title
Time to clinical worsening during iNO withdrawal for up to 8 weeks
Description
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)
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
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.
Description
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)
Time Frame
8 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ashika Ahmed, MD
Organizational Affiliation
Bellerophon Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Bluhm Cardiovascular Institute, Clinical Trials Unit
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Peter Lougheed Centre
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T1Y6J4
Country
Canada
Facility Name
Toronto General Hospital, University Health Network
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Study of PAH Subjects With LTOT Use That Have Demonstrated Improved Exercise Tolerance With the Use of Inhaled Nitric

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