Clinical trial NCT00705133: Treprostinil Therapy For Patients With Interstitial Lung Disease And Severe Pulmonary Arterial Hypertension
Official title: Using Either Intravenous (IV) or Subcutaneous (SQ) Treprostinil to Treat Pulmonary Hypertension Related to Underlying Interstitial Lung Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pulmonary Arterial Hypertension
Interstitial Lung Disease
Idiopathic Pulmonary Fibrosis
Keywords: pulmonary hypertension
pulmonary fibrosis
interstitial lung disease
Lead sponsor: University of California, Los Angeles
Sponsor class: OTHER
Collaborators: name: United Therapeutics
First posted: 2008-06-25
Enrollment: 15
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Our hypothesis is that IV or SQ Treprostinil can improve 6 minute walk distance, hemodynamics and quality of life in patients with interstitial lung disease and severe secondary pulmonary arterial hypertension.

Detailed description: Patients with pulmonary hypertension (PH) complicating pulmonary fibrosis are at increased risk of death. There are no therapies proven to be effective in this population, targeting the pulmonary hypertension. The purpose of this study is to evaluate parenteral treprostinil in an open-label fashion in patients with pulmonary fibrosis and an advanced PH phenotype.

Eligibility criteria: Inclusion Criteria:

Eligible subjects must have IPF and severe pulmonary arterial hypertension (PAH) documented on standard of care right-heart catheterization (RHC) and planned to receive therapy with treprostinil as recommended by the treating physician.

1. All subjects must have high resolution CT scan (HRCT) diagnostic of IPF (performed as part of standard of care evaluation) or if available, biopsy proven histological usual interstitial pneumonia (UIP).
2. Severe pulmonary arterial hypertension defined as a resting mean pulmonary artery pressure (mPAP) \> 35 mm Hg; AND pulmonary vascular resistance (PVR) \> 3 woods-units; AND pulmonary capillary wedge pressure (PCWP) \< 18 mm Hg by right-heart catheterization (RHC) performed as part of standard of care evaluation.
3. All subjects must be planned to receive treprostinil therapy as recommended by their treating physician.

Exclusion Criteria:

1. Acute or chronic impairment other than dyspnea (e.g. angina pectoris, intermittent claudication) limiting the ability to perform standard of care six-minute walk tests (6MWT).
2. Six-minute walk distance (6MWD) \< 50 meters at screening or baseline standard of care evaluations
3. Standard of care pulmonary function test (PFT) showing forced expiratory volume in one second (FEV1)/ forced vital capacity (FVC) ratio \< 0.65
4. Standard of care pulmonary function test (PFT) showing a residual volume \>120% predicted
5. Standard of care high-resolution chest computed tomography (HRCT) showing emphysema extent \> 30%
6. Any investigational therapy as part of a clinical trial for any indication with 30 days before screening
7. Change in dose of treatment for IPF - investigational agent (gamma interferon-1b, pirfenidone, etanercept, and any other investigational agent intended to treat IPF), corticosteroids, or cytotoxic agents, within 30 days before screening. That is, subjects can be on any of these agents provided the dose is stable for at least 30 days prior to enrollment.
8. Current treatment for pulmonary hypertension with other prostaglandins (epoprostenol or iloprost)
9. Change in dose of treatment for PAH - (bosentan, sitaxsentan, ambrisentan, tadalafil, sildenafil, vardenafil, calcium channel blockers, nitrates, digitalis), within 30 days before screening. That is, subjects can be on any of these agents provided the dose is stable for at least 30 days prior to enrollment
10. Pulmonary rehabilitation initiated within 30 days of baseline.

Primary outcomes:
measure: 6 Minute Walk Distance
timeFrame: 3 months
Secondary outcomes:
measure: Pulmonary Vascular Resistance
timeFrame: 3 months
measure: SF-36 Quality of Life
timeFrame: 3 months
measure: Brain Natriuretic Peptide
timeFrame: 3 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: David Geffen School of Medicine, UCLA
state: California
Study officials:
affiliation: David Geffen School of Medicine, UCLA
name: Rajan Saggar, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: David Geffen School of Medicine, UCLA
name: David Zisman, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Saggar R, Khanna D, Vaidya A, Derhovanessian A, Maranian P, Duffy E, Belperio JA, Weigt SS, Dua S, Shapiro SS, Goldin JG, Abtin F, Lynch JP 3rd, Ross DJ, Forfia PR, Saggar R. Changes in right heart haemodynamics and echocardiographic function in an advanced phenotype of pulmonary hypertension and right heart dysfunction associated with pulmonary fibrosis. Thorax. 2014 Feb;69(2):123-9. doi: 10.1136/thoraxjnl-2013-204150.