Trial Outcomes & Findings for A Study of Treprostinil to Treat Interstitial Lung Disease Pulmonary Hypertension (NCT NCT05564637)

NCT ID: NCT05564637

Last Updated: 2025-12-03

Results Overview

Cardiac output reserve (Qc) is the difference between the heart's maximum output during exercise and its resting output. This was measured using the direct Fick method (L/min).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

29 participants

Primary outcome timeframe

Baseline, approximately 35 minutes

Results posted on

2025-12-03

Participant Flow

29 subjects were consented of which 5 screen failed prior to study group assignment.

Participant milestones

Participant milestones
Measure
Healthy Volunteers
Healthy volunteers will undergo RHC while exercising and an optional muscle biopsy. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter will be inserted into a vein and artery in order to take the measurements and this will be conducted while exercising on a supine cycle ergometer. Various measurements will be taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue will be collected and analyzed for the ability of the cells to utilize oxygen.
PAH-ILD Patients
Patients diagnosed with PAH-ILD will undergo RHC while exercising, receive inhaled treprostinil, and undergo an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil will be administered during the RHC procedure and patients will be initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter will be inserted into a vein and artery in order to take the measurements and this will be conducted while exercising on a supine cycle ergometer. Various measurements will be taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue will be collected and analyzed for the ability of the cells to utilize oxygen.
Overall Study
STARTED
1
23
Overall Study
COMPLETED
1
22
Overall Study
NOT COMPLETED
0
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of Treprostinil to Treat Interstitial Lung Disease Pulmonary Hypertension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
PAH-ILD Patients
n=23 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Total
n=23 Participants
Total of all reporting groups
Age, Continuous
72.5 years
STANDARD_DEVIATION 8 • n=3 Participants
72.5 years
STANDARD_DEVIATION 8 • n=6 Participants
Sex: Female, Male
Female
10 Participants
n=3 Participants
0 Participants
n=3 Participants
10 Participants
n=6 Participants
Sex: Female, Male
Male
13 Participants
n=3 Participants
0 Participants
n=3 Participants
13 Participants
n=6 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=3 Participants
0 Participants
n=3 Participants
0 Participants
n=6 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
18 Participants
n=3 Participants
0 Participants
n=3 Participants
18 Participants
n=6 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
5 Participants
n=3 Participants
0 Participants
n=3 Participants
5 Participants
n=6 Participants
Race (NIH/OMB)
American Indian or Alaska Native
1 Participants
n=3 Participants
0 Participants
n=3 Participants
1 Participants
n=6 Participants
Race (NIH/OMB)
Asian
0 Participants
n=3 Participants
0 Participants
n=3 Participants
0 Participants
n=6 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=3 Participants
0 Participants
n=3 Participants
0 Participants
n=6 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=3 Participants
0 Participants
n=3 Participants
0 Participants
n=6 Participants
Race (NIH/OMB)
White
17 Participants
n=3 Participants
0 Participants
n=3 Participants
17 Participants
n=6 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=3 Participants
0 Participants
n=3 Participants
0 Participants
n=6 Participants
Race (NIH/OMB)
Unknown or Not Reported
5 Participants
n=3 Participants
0 Participants
n=3 Participants
5 Participants
n=6 Participants
Region of Enrollment
United States
23 participants
n=3 Participants
23 participants
n=6 Participants

PRIMARY outcome

Timeframe: Baseline, approximately 35 minutes

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Cardiac output reserve (Qc) is the difference between the heart's maximum output during exercise and its resting output. This was measured using the direct Fick method (L/min).

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Cardiac Output Reserve (Qc)
Baseline
3.3 Liters/Min
Standard Deviation 2.1
Cardiac Output Reserve (Qc)
Approximately 35 minutes
3.8 Liters/Min
Standard Deviation 1.8

SECONDARY outcome

Timeframe: Baseline, approximately 35 minutes

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Ventilatory efficiency was assessed as a ratio between the ventilation (VE) and carbon dioxide production (VCO2) measured during the RHC procedure. VE/VCO2 evaluates how efficiently a person breathes during exercise.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=16 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Ventilatory Efficiency (VE/VCO2)
Baseline
50.4 Ratio
Standard Deviation 10.6
Ventilatory Efficiency (VE/VCO2)
Approximately 35 Minutes
51.2 Ratio
Standard Deviation 11.2

SECONDARY outcome

Timeframe: Baseline, 3-months

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Participants severity of heart failure symptoms was classified according to the NHYA functional class (I-IV). Lower classifications indicate lower severity of heart failure symptoms.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
New York Heart Association (NYHA) Functional Class
NYHA III
6 Participants
New York Heart Association (NYHA) Functional Class
NYHA IV
6 Participants
New York Heart Association (NYHA) Functional Class
NYHA I
1 Participants
New York Heart Association (NYHA) Functional Class
NYHA II
9 Participants

SECONDARY outcome

Timeframe: Baseline; after 30 minutes of rest

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Resting pulmonary vascular resistance was measured in Wood units (mm Hg/L/min) during the RHC procedure. Resting pulmonary vascular resistance is the resistance against blood flow from the pulmonary veins to the left atrium of the heart.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Resting Pulmonary Vascular Resistance
Baseline
5.4 mm Hg/L/min
Standard Deviation 2.1
Resting Pulmonary Vascular Resistance
After 30 minutes of Rest
3.1 mm Hg/L/min
Standard Deviation 1.4

SECONDARY outcome

Timeframe: Baseline; approximately 35 minutes

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Exercise pulmonary vascular resistance was measured in Wood units (mm Hg/L/min) during the RHC procedure.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Exercise Pulmonary Vascular Resistance
Baseline
4.3 mm Hg/L/min
Standard Deviation 2.1
Exercise Pulmonary Vascular Resistance
Approximately 35 minutes
2.2 mm Hg/L/min
Standard Deviation 1.2

SECONDARY outcome

Timeframe: Baseline; after 30 minutes of rest

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Resting pulmonary artery pressure (mmHg) was measured during the RHC procedure. Pulmonary artery pressure is the pressure within the pulmonary arteries, which transport deoxygenated blood from the right side of the heart to the lungs for oxygenation.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Resting Pulmonary Artery Pressure
Baseline
36.0 mmHg
Standard Deviation 7.3
Resting Pulmonary Artery Pressure
After 30 minutes of Rest
25.1 mmHg
Standard Deviation 5.2

SECONDARY outcome

Timeframe: Baseline; approximately 35 minutes

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Exercise Pulmonary artery pressure (mmHg) was measured during the RHC procedure. Pulmonary artery pressure is the pressure within the pulmonary arteries, which transport deoxygenated blood from the right side of the heart to the lungs for oxygenation.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Exercise Pulmonary Artery Pressure
Baseline
50.4 mmHg
Standard Deviation 9.3
Exercise Pulmonary Artery Pressure
Approximately 35 Minutes
40.6 mmHg
Standard Deviation 9.0

SECONDARY outcome

Timeframe: Baseline; after 30 minutes of rest

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Resting right atrial pressure (mmHg) was measured during the RHC procedure. Right atrial pressure is a measurement that reflects the amount of blood returning to the heart and the heart's ability to pump blood into the arterial system.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Resting Right Atrial Pressure
Baseline
7.9 mmHg
Standard Deviation 3.4
Resting Right Atrial Pressure
After 30 Minutes of Rest
6.2 mmHg
Standard Deviation 3.1

SECONDARY outcome

Timeframe: Baseline; approximately 35 minutes

Population: Only 1 participant was enrolled in the healthy volunteers arm. Based on the low enrollment number in the healthy volunteers arm, no data is reported for the 1 subject in order to protect and maintain participant privacy/confidentiality.

Exercise right atrial pressure (mmHg) was measured during the RHC procedure. Right atrial pressure is a measurement that reflects the amount of blood returning to the heart and the heart's ability to pump blood into the arterial system.

Outcome measures

Outcome measures
Measure
PAH-ILD Patients
n=22 Participants
Patients diagnosed with PAH-ILD underwent RHC while exercising, received inhaled treprostinil, and underwent an optional muscle biopsy. Treprostinil: 30mcg (5 breaths) of inhaled treprostinil was administered during the RHC procedure and patients were initiated on outpatient inhaled treprostinil therapy for 3-months with gradual up titration to target dose as per standard clinical practice. Right Heart Catheterization (RHC) while exercising: A procedure to measure how well or poorly your heart is pumping and to measure the pressures in the heart and lungs. A catheter was inserted into a vein and artery in order to take the measurements and this was conducted while exercising on a supine cycle ergometer. Various measurements were taken during periods of rest and exercise. Optional Muscle Biopsy: A sample of muscle tissue were collected and analyzed for the ability of the cells to utilize oxygen.
Healthy Volunteers
Healthy volunteers underwent RHC while exercising and an optional muscle biopsy.
Exercise Right Atrial Pressure
Baseline
16.1 mmHg
Standard Deviation 6.3
Exercise Right Atrial Pressure
Approximately 35 Minutes
12.9 mmHg
Standard Deviation 6.0

Adverse Events

Healthy Volunteers

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

PAH-ILD Patients

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Yogesh Reddy, M.B.B.S.

Mayo Clinic

Phone: 507-284-2904

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place