Clinical Investigation Into Inhaled Treprostinil Sodium in Patients With Severe Pulmonary Arterial Hypertension (PAH)

NCT ID: NCT00147199

Last Updated: 2024-01-02

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

235 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-06-30

Study Completion Date

2007-10-31

Brief Summary

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This is a double-blind placebo-controlled clinical investigation into the efficacy and tolerability of inhaled treprostinil in patients with severe pulmonary arterial hypertension. The primary outcome is the change in 6-minute walk distance from baseline to week 12.

Detailed Description

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Patients who have been on a stable dose of 125 mg twice daily (bid) of bosentan or any stable dose of sildenafil for at least three months prior to study start were randomized to either treprostinil inhalation solution or matching placebo.

Administration of study medication was performed by inhalation with the OPTINEB™ ultrasonic nebulizer.

The proposed dosing regimen was four times daily-upon awakening, at midday, evening (dinner time) and bedtime.

After a patient has completed the twelve-week study period, they were given the option of enrolling into an open-label extension study.

Conditions

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Pulmonary Hypertension

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Inhaled treprostinil

0.9 mg/mL treprostinil for inhalation supplied in 2.9mL ampoules for use in ultra sonic nebulizer

Group Type EXPERIMENTAL

Inhaled treprostinil

Intervention Type DRUG

Doses are titrated to 9 breaths four times daily. Each breath produces an 18 mcg dose of inhaled treprostinil.

Placebo

Placebo inhalation solution for use in ultrasonic nebulizer

Group Type PLACEBO_COMPARATOR

Placebo inhalation solution

Intervention Type DRUG

Doses are titrated to 9 breaths four times daily.

Interventions

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Inhaled treprostinil

Doses are titrated to 9 breaths four times daily. Each breath produces an 18 mcg dose of inhaled treprostinil.

Intervention Type DRUG

Placebo inhalation solution

Doses are titrated to 9 breaths four times daily.

Intervention Type DRUG

Other Intervention Names

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Tyvaso Placebo

Eligibility Criteria

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Inclusion Criteria

* Clinically stable, pulmonary arterial hypertension diagnosed as either idiopathic or familial PAH, collagen vascular disease associated PAH, HIV PAH, or PAH induced by anorexigens, New York Heart Association (NYHA) Class III or Class IV.
* Been on a stable dose of 125 mg twice daily (bid) of bosentan OR any stable dose of sildenafil for at least three months prior to study start
* An unencouraged six minute walk test (6MWT) of between 200 and 450 meters at screening
* Cardiac catheterization within the past 13 months consistent with PAH, specifically mean pulmonary artery pressure (PAPm) ≥25 mmHg (at rest), pulmonary capillary wedge pressure (PCWP) (or left ventricular end diastolic pressure) ≤15 mmHg, and pulmonary vascular resistance (PVR) \>3 mmHg/L/min
* Within the past 12 months, patients must have had a chest radiograph consistent with the diagnosis of PAH
* Willing and able to follow all study procedures

* Have had any change in or discontinued any PAH medication within the last three months, including but not limited to endothelin receptor antagonist (ERA), or calcium channel blockers (CCB) (with the exception of anticoagulants)
* Have received any prostanoid within the 30 days before screening or are scheduled to receive any during the course of the study
* Have received any investigational medication within 30 days prior to the start of this study or are scheduled to receive another investigational drug during the course of this study
* Have a known intolerance to any drug, especially to treprostinil sodium or prostanoids
* Have an increased risk of hemorrhage
* Have a new type of chronic therapy (e.g., a different category of vasodilator, diuretic) for PAH added within the last month, except anticoagulants
* Have any musculoskeletal disease or any other disease that would limit ambulation.

Exclusion Criteria

* Considering pregnancy, are pregnant and/or lactating
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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United Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

University of Arizona

Tucson, Arizona, United States

Site Status

UCSD Medical center

La Jolla, California, United States

Site Status

UCLA Medical Center

Torrance, California, United States

Site Status

University of Colorado

Aurora, Colorado, United States

Site Status

Orlando Heart Center

Orlando, Florida, United States

Site Status

Kansas University Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Tufts Medical Center

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Bethe Israel Medical Center

New York, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Allegheny General Hospital

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

UTSW Medical Center

Dallas, Texas, United States

Site Status

Universitatsklinikfur Innere Medizin II

Wein, Vienna, Austria

Site Status

Medical University Graz

Graz, , Austria

Site Status

Universite Libre de Bruxelles

Brussels, , Belgium

Site Status

University Hospital Gasthuisburg

Leuven, , Belgium

Site Status

Hospital Antoine Beclere

Paris, , France

Site Status

Univesitatsklinikum Giessen und Marburg GmbH

Geißen, , Germany

Site Status

Pulmonary Hypertension Unit

Dublin, , Ireland

Site Status

Rambam Medical Center

Haifa, , Israel

Site Status

The Pulmonary Institute

Jerusalem, , Israel

Site Status

The Pulmonary Institute

Petach Tikvah 49100, , Israel

Site Status

Instituto Malattie dell'Apparato Vascolare

Bologna, , Italy

Site Status

University of Barcelona

Barcelona, , Spain

Site Status

Papworth Hospital

Cambridge, , United Kingdom

Site Status

Scottish Pulmonary Vascular Unit

Glasgow GII 6NT, , United Kingdom

Site Status

Royal Fee Hospital

London, , United Kingdom

Site Status

Countries

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United States Austria Belgium France Germany Ireland Israel Italy Spain United Kingdom

References

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McLaughlin VV, Benza RL, Rubin LJ, Channick RN, Voswinckel R, Tapson VF, Robbins IM, Olschewski H, Rubenfire M, Seeger W. Addition of inhaled treprostinil to oral therapy for pulmonary arterial hypertension: a randomized controlled clinical trial. J Am Coll Cardiol. 2010 May 4;55(18):1915-22. doi: 10.1016/j.jacc.2010.01.027.

Reference Type DERIVED
PMID: 20430262 (View on PubMed)

Related Links

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http://www.phassociation.org

The mission of the Pulmonary Hypertension Association is to seek a cure, and to provide hope, support and education, and to promote awareness and to advocate for the pulmonary hypertension community.

Other Identifiers

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LRX-TRIUMPH 001

Identifier Type: -

Identifier Source: org_study_id

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