Trial Outcomes & Findings for Transition From Injectable Prostacyclin Medication to Inhaled Prostacyclin Medication (NCT NCT01268553)

Results Overview

The number of adverse events will be recorded at transition, 4 weeks, and 12 weeks.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

6 participants

Primary outcome timeframe

12 Weeks

Results posted on

2017-09-01

Participant Flow

Participant milestones

Participant milestones
Measure	Open Label Single Arm Group World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

3 screen failures

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open Label Single Arm Group n=9 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
Age, Continuous	50 years STANDARD_DEVIATION 17 • n=9 Participants
Sex: Female, Male Female	8 Participants n=9 Participants
Sex: Female, Male Male	1 Participants n=9 Participants
Region of Enrollment United States	9 Participants n=9 Participants
pulmonary vascular resistance	4.2 Wood Units STANDARD_DEVIATION 2.4 • n=6 Participants • 3 screen failures
6-minute walk distance	334 meters STANDARD_DEVIATION 132 • n=6 Participants • 3 screen failures
peak oxygen consumption	1.0 L/min STANDARD_DEVIATION 0.2 • n=6 Participants • 3 screen failures
ratio of minute ventilation to CO2 output	32 ratio STANDARD_DEVIATION 2 • n=6 Participants • 3 screen failures
The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR)	6.8 units on a scale STANDARD_DEVIATION 6 • n=6 Participants • 3 screen failures

PRIMARY outcome

Timeframe: 12 Weeks

Population: Number of patients weaned off of their parenteral prostanoids without adverse events

The number of adverse events will be recorded at transition, 4 weeks, and 12 weeks.

Outcome measures

Outcome measures
Measure	Open Label Single Arm Group n=6 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
Number of Participants Without Adverse Events	6 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: Patients weaned off of their parenteral prostanoids without Clinical Worsening

Clinical worsening is defined as any of the following: * All-cause mortality * Nonelective hospital stay for PAH (with predefined criteria, usually for initiation of intravenous prostanoids, lung transplantation, or septostomy) * Disease progression defined as a reduction from baseline in the 6MW test by 15%, confirmed by 2 studies done within 2 weeks plus worsening functional class

Outcome measures

Outcome measures
Measure	Open Label Single Arm Group n=6 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
Number of Participants Without Clinical Worsening	6 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: Mean change in 6-minute walk distance

Change in 6-minute walk distance from baseline to 12 weeks.

Outcome measures

Outcome measures
Measure	Open Label Single Arm Group n=6 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
Change in 6-minute Walk Distance	20 meters Standard Deviation 58

SECONDARY outcome

Timeframe: 12 weeks

Ventilatory efficiency measured with cardiopulmonary exercise testing

Outcome measures

Outcome measures
Measure	Open Label Single Arm Group n=6 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
VE/VCO	1.2 ratio Standard Deviation 2.0

SECONDARY outcome

Timeframe: 12 q=weeks

The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) is a disease specific patient-reported outcome measure which assesses quality of life of patients with pulmonary hypertension (PH). QoL scores (total) range from 0-25, with higher scores indicating worse quality of life

Outcome measures

Outcome measures
Measure	Open Label Single Arm Group n=6 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
CAMPHOR: Cambridge Pulmonary Hypertension Outcome Review; Construct = Quality of Life	-0.5 units on a scale Standard Deviation 3.4

SECONDARY outcome

Timeframe: 12 weeks

N-terminal pro BNP level

Outcome measures

Outcome measures
Measure	Open Label Single Arm Group n=6 Participants World Health Organization (WHO) group 1 PAH patients receiving long-term parenteral prostanoids
N-terminal Pro BNP Level	14 pg/mL Standard Deviation 52

Adverse Events

Open Label Single Arm Group

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

Dr. Ronald Oudiz

Los Angeles Biomedical research INst at Harbor-UCLA

Phone: 310-222-3560

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place