Trial Outcomes & Findings for Iloprost Power 15 in Pulmonary Arterial Hypertension (NCT NCT00709956)

Last Updated: 2025-02-04

Results Overview

The 6-minute walk test was performed 20-40 minutes after treatment. This was a non-encouraged test (the person conducting the test did not encourage the patient to walk farther or faster) that measured the distance covered over a 6-minute walk. It was conducted by a trained member of the site staff who was listed on the site's delegation of authority sheet. For patients who had never performed a 6-minute walk test previously, a training test was requested before the qualifying tests for randomization.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

64 participants

Primary outcome timeframe

Study day 2 or study day 3

Results posted on

2025-02-04

Participant Flow

On Day 1 all patients received a single inhalation dose of placebo. Patients who satisfied the selection criteria in the single-blind period of the study were entered into the double-blind period to receive iloprost 5 μg and matching placebo (Days 2 and 3) in a single dose, two-period crossover design.

Of the 70 randomized patients, 64 patients entered the double-blind period, (placebo/iloprost P15, 33 patients and iloprost P15/placebo, 31 patients). 1 patient in the iloprost P15/placebo treatment sequence was excluded from the per-protocol analysis which was defined as the primary analysis.

Participant milestones

Participant milestones
Measure	Iloprost (5µg) / Placebo Single dose double-blind active iloprost (5µg) on study day 2 followed by single dose double-blind placebo on study day 3	Placebo / Iloprost (5 µg) Single dose double-blind placebo on study day 2 followed by single dose double-blind active iloprost (5µg) on study day 3
Overall Study STARTED	33	37
Overall Study Double-blind Crossover-period	31	33
Overall Study COMPLETED	30	33
Overall Study NOT COMPLETED	3	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Iloprost (5µg) / Placebo Single dose double-blind active iloprost (5µg) on study day 2 followed by single dose double-blind placebo on study day 3	Placebo / Iloprost (5 µg) Single dose double-blind placebo on study day 2 followed by single dose double-blind active iloprost (5µg) on study day 3
Overall Study Withdrawal by Subject	1	1
Overall Study Inclusion/exclusion criteria violation	2	3

Baseline Characteristics

Iloprost Power 15 in Pulmonary Arterial Hypertension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Iloprost (5µg) / Placebo n=31 Participants Single dose double-blind active iloprost (5µg) on study day 2 followed by single dose double-blind placebo on study day 3	Placebo / Iloprost (5 µg) n=33 Participants Single dose double-blind placebo on study day 2 followed by single dose double-blind active iloprost (5µg) on study day 3	Total n=64 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	22 Participants n=5 Participants	24 Participants n=7 Participants	46 Participants n=5 Participants
Age, Categorical >=65 years	9 Participants n=5 Participants	9 Participants n=7 Participants	18 Participants n=5 Participants
Age, Continuous	55.5 years STANDARD_DEVIATION 13.57 • n=5 Participants	55.8 years STANDARD_DEVIATION 16.40 • n=7 Participants	55.7 years STANDARD_DEVIATION 14.98 • n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants	28 Participants n=7 Participants	51 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	5 Participants n=7 Participants	13 Participants n=5 Participants
Region of Enrollment United States	28 participants n=5 Participants	30 participants n=7 Participants	58 participants n=5 Participants
Region of Enrollment Austria	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Germany	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants

PRIMARY outcome

Timeframe: Study day 2 or study day 3

Population: The analysis was per protocol, 64 patients started double blind phase of study, 63 completed.

Outcome measures

Outcome measures
Measure	6MWD After Placebo Treatment n=63 Participants	6MWD After Iloprost (5 µg) Treatment n=63 Participants
6-minute-walk Distance (6MWD)	330.0 meters 95% Confidence Interval 22.84 • Interval 322.3 to 337.7	328.6 meters 95% Confidence Interval 55.28 • Interval 320.9 to 336.2

SECONDARY outcome

Timeframe: Study day 2 or study day 3

Population: The analysis was per protocol, 64 patients started double blind phase of study, 63 completed.

The Borg scale is a category-ratio scale, commonly used to evaluate the effects of exercise on dyspnea. The original and modified scales have ratio properties ranging from 0 = nothing at all to 10 = very, very severe, with descriptors from 0 to 10. Descriptors have been modified by others so that 10 has been labeled "extremely severe," or "the worst possible dyspnea imaginable." Reliability and validity have been reported in a general population and in patients with PAH as well as other respiratory conditions.

Outcome measures

Outcome measures
Measure	6MWD After Placebo Treatment n=63 Participants	6MWD After Iloprost (5 µg) Treatment n=63 Participants
Borg Dyspnea Score	3.5 scores on a scale Standard Deviation 2.23	3.4 scores on a scale Standard Deviation 2.25

Adverse Events

Iloprost (5µg) / Placebo

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

Placebo / Iloprost (5 µg)

Serious events: 1 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Iloprost (5µg) / Placebo n=31 participants at risk Single dose double-blind active iloprost (5µg) on study day 2 followed by single dose double-blind placebo on study day 3	Placebo / Iloprost (5 µg) n=33 participants at risk Single dose double-blind placebo on study day 2 followed by single dose double-blind active iloprost (5µg) on study day 3
Respiratory, thoracic and mediastinal disorders Pulmonary arterial hypertension	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	3.0% 1/33 • Number of events 1 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Cardiac disorders Right ventricular failure	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	3.0% 1/33 • Number of events 1 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.

Other adverse events

Additional Information

Laila Rouault, M.D.

Actelion

Phone: + 41 61 565 8128

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Iloprost (5µg) / Placebo n=31 participants at risk Single dose double-blind active iloprost (5µg) on study day 2 followed by single dose double-blind placebo on study day 3	Placebo / Iloprost (5 µg) n=33 participants at risk Single dose double-blind placebo on study day 2 followed by single dose double-blind active iloprost (5µg) on study day 3
Respiratory, thoracic and mediastinal disorders Cough	12.9% 4/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	12.1% 4/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Gastrointestinal disorders Diarrhoea	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	6.1% 2/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Nervous system disorders Dizziness	6.5% 2/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	12.1% 4/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Respiratory, thoracic and mediastinal disorders Dysphonia	6.5% 2/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	0.00% 0/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Respiratory, thoracic and mediastinal disorders Dyspnoea	3.2% 1/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	9.1% 3/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Vascular disorders Flushing	12.9% 4/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	15.2% 5/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Nervous system disorders Headache	9.7% 3/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	9.1% 3/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Musculoskeletal and connective tissue disorders Muscle tightness	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	6.1% 2/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Gastrointestinal disorders Nausea	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	6.1% 2/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	3.2% 1/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	9.1% 3/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Musculoskeletal and connective tissue disorders Pain in extremity	6.5% 2/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	0.00% 0/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
General disorders Pyrexia	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	6.1% 2/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Skin and subcutaneous tissue disorders Rash	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	6.1% 2/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	0.00% 0/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	9.1% 3/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.
Respiratory, thoracic and mediastinal disorders Throat irritation	16.1% 5/31 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.	3.0% 1/33 Due to data capture design constraints the time of adverse event (AE) onset was not collected, therefore the potential to assign potential causality to active treatment or placebo was not assessed. AE's can only be reported as shown, as prospectively planned in the protocol submitted to FDA. Includes all patients entered in the double-blind phase.