Trial Outcomes & Findings for Inhaled Treprostinil in Participants With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease (PH-COPD) (NCT NCT03794583)

Last Updated: 2025-12-11

Results Overview

An adverse event (AE) can be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. Treatment-emergent was defined as any AE occurring/worsening at any time after a participant was exposed to study drug up until 7 days after the last dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

41 participants

Primary outcome timeframe

Up to 4 years

Results posted on

2025-12-11

Participant Flow

41 participants, who either successfully completed or were administratively terminated due to the COVID-19 pandemic from Study RIN-PH-304 (NCT03496623), chose to enroll in Study RIN-PH-305 (NCT03794583) open-label extension study.

Participant milestones

Participant milestones
Measure	Treprostinil Participants received up to 90 micrograms (μg) of treprostinil for inhalation four times daily (QID), for up to 4 years
Overall Study STARTED	41
Overall Study Received At Least 1 Dose of Study Drug	41
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	41

Reasons for withdrawal

Reasons for withdrawal
Measure	Treprostinil Participants received up to 90 micrograms (μg) of treprostinil for inhalation four times daily (QID), for up to 4 years
Overall Study Death	8
Overall Study Study Terminated by Sponsor	24
Overall Study Withdrawal by Subject	9

Baseline Characteristics

Inhaled Treprostinil in Participants With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease (PH-COPD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treprostinil n=41 Participants Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Race (NIH/OMB) White	31 Participants n=237 Participants
Race (NIH/OMB) More than one race	2 Participants n=237 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=237 Participants
Age, Continuous	68.24 years STANDARD_DEVIATION 7.35 • n=237 Participants
Sex: Female, Male Female	17 Participants n=237 Participants
Sex: Female, Male Male	24 Participants n=237 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=237 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	39 Participants n=237 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=237 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=237 Participants
Race (NIH/OMB) Asian	0 Participants n=237 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=237 Participants
Race (NIH/OMB) Black or African American	8 Participants n=237 Participants

PRIMARY outcome

Timeframe: Up to 4 years

Population: Safety population included all participants who received at least 1 dose of inhaled treprostinil.

Outcome measures

Outcome measures
Measure	Treprostinil n=41 Participants Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	35 Participants

SECONDARY outcome

Timeframe: Baseline, Week 6

Population: All participants set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure and 'Number analyzed' = participants evaluable at the specified timeframe.

6MWD was calculated at peak exposure (10 to 60 minutes after dosing). 6-minute walk test (6MWT) was performed by standardized procedures for all participants. Participants were asked to walk a set course for 6 minutes (timed) and the distance walked (in meters) was recorded.

Outcome measures

Outcome measures
Measure	Treprostinil n=38 Participants Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Change From Baseline to Week 6 in 6-Minute Walk Distance (6MWD) Change at Week 6	15 meters Standard Deviation 43.58
Change From Baseline to Week 6 in 6-Minute Walk Distance (6MWD) Baseline	226.5 meters Standard Deviation 88.91

SECONDARY outcome

Timeframe: Baseline, Week 6

The Borg Dyspnea Score was a 11-point scale rating the maximum level of dyspnea experienced during the 6MWT. Scores range from 0 (no dyspnea at all) to 10 (very, very severe dyspnea), with lower scores indicating less exertion (a better outcome). The Borg Dyspnea Score was to be evaluated immediately after the 6MWT.

Outcome measures

Outcome measures
Measure	Treprostinil n=38 Participants Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Change From Baseline to Week 6 in Borg Dyspnea Score Baseline	5.03 score on a scale Standard Deviation 2.57
Change From Baseline to Week 6 in Borg Dyspnea Score Change at Week 6	0.38 score on a scale Standard Deviation 1.94

SECONDARY outcome

Timeframe: Baseline, Week 6

The NT-proBNP concentration is a biomarker associated with changes in right heart morphology and function. Improvement is defined as a decrease in the NT-proBNP plasma concentration.

Outcome measures

Outcome measures
Measure	Treprostinil n=40 Participants Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Change From Baseline to Week 6 in N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Baseline	1279.6 nanogram/liter (ng/L) Standard Deviation 2080.19
Change From Baseline to Week 6 in N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) Change at Week 6	-155.55 nanogram/liter (ng/L) Standard Deviation 558.96

Adverse Events

Treprostinil

Serious events: 13 serious events

Other events: 22 other events

Deaths: 8 deaths

Serious adverse events

Serious adverse events
Measure	Treprostinil n=41 participants at risk Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	12.2% 5/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	9.8% 4/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Respiratory, thoracic and mediastinal disorders Pulmonary hypertension	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Respiratory, thoracic and mediastinal disorders Respiratory failure	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Blood and lymphatic system disorders Anaemia	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Cardiac disorders Acute myocardial infarction	4.9% 2/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Cardiac disorders Cardiac arrest	4.9% 2/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Cardiac disorders Myocardial infarction	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Cardiac disorders Right ventricular failure	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
General disorders Sudden death	4.9% 2/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Infections and infestations COVID-19 pneumonia	4.9% 2/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Infections and infestations COVID-19	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Infections and infestations Cellulitis	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Nervous system disorders Seizure	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Nervous system disorders Syncope	2.4% 1/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.

Other adverse events

Other adverse events
Measure	Treprostinil n=41 participants at risk Participants received up to 90 μg of treprostinil for inhalation QID, for up to 4 years
Infections and infestations COVID-19	12.2% 5/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Musculoskeletal and connective tissue disorders Back pain	9.8% 4/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Nervous system disorders Headache	14.6% 6/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Psychiatric disorders Anxiety	7.3% 3/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Respiratory, thoracic and mediastinal disorders Cough	19.5% 8/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	9.8% 4/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.
Respiratory, thoracic and mediastinal disorders Dyspnoea	9.8% 4/41 • Up to 4 years Safety population included all participants who received at least 1 dose of inhaled treprostinil.

Additional Information

United Therapeutics Global Medical Information

United Therapeutics

Phone: 1-240-821-1881

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Institution and/or Principal Investigator agree not to publish or publicly present any results of the Study without the prior written consent of Sponsor, not to be unreasonably withheld or delayed. Institution and/or Principal Investigator further agree to provide Sponsor with drafts of any such publication or presentation for review and approval no less than 30 days prior to submission for publication or the date of public presentation.
Publication restrictions are in place

Restriction type: OTHER