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Phase 3 N=235 Randomized Quadruple-blind Treatment

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

Pulmonary Hypertension

Enrolled (actual)
235
Serious AEs
9.4%
Results posted
Aug 2013
Primary outcome: Primary: Peak 6-minute Walk Distance — 366.0; 360.0; 21.6; 3.0 meters — p=0.00044

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Inhaled treprostinil (Drug); Placebo inhalation solution (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
United Therapeutics
Primary completion
Oct 2007

Outcome Measures

OutcomeResultp-value
PRIMARY
Peak 6-minute Walk Distance
366.0; 360.0; 21.6; 3.0 0.00044 sig
SECONDARY
Clinical Worsening Events
4; 6
SECONDARY
Borg Dyspnea Score
3.68; 3.91; 0.0; 0.0 0.623
SECONDARY
New York Heart Association (NYHA) Functional Classification
22; 22; 88; 93; 5; 5 0.807
SECONDARY
Trough 6MWD at Week 12
364.0; 365.0; 12.4; 0.8 0.007 sig
SECONDARY
Peak 6MWD at Week 6
375; 367; 21.6; 3.0 0.0003 sig
SECONDARY
Quality of Life (Minnesota Living With Heart Failure)
-3.0; 0.0; -1.4; 0.0; 0.0; 0.0 0.027 sig
SECONDARY
Change in Signs and Symptoms of PAH
0; 0
SECONDARY
N-terminal Pro-B-Type Natriuretic Peptide (NT Pro-BNP)
377; 756; -57; 40 0.001 sig

Summary

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.

Eligibility Criteria

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

Exclusion Criteria

  • Considering pregnancy, are pregnant and/or lactating
  • PAH due to conditions other than noted in the above inclusion criteria.
  • 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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00147199). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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