A Phase 2, Placebo Controlled, Double-Blind, Randomized, Clinical Study to Determine Safety, Tolerability and Efficacy of Pulsed, Inhaled Nitric Oxide (iNO) Versus Placebo as Add-On Therapy in Symptomatic Subjects With Pulmonary Arterial Hypertension (PAH)

Study ID
STU 082012-039

Cancer Related
No

Healthy Volunteers
No

Study Sites

Contact
Jacqueline Quivers
214-645-6489
jacqueline.quivers@utsouthwestern.edu

Principal Investigator
Sonja Bartolome

Summary

This is a phase 2, placebo-controlled, double-blind, randomized, clinical study to determine safety, tolerability and efficacy of pulsed ino versus placebo as add-on therapy in symptomatic subjects with PaH. The study will be conducted in two parts.
all subjects will be treated by means of a blinded inopulse DS delivery device. all subjects will change study drug minicylinders approximately every 24 hours

Participant Eligibility

1. Signed informed consent form (ICF) (and assent as appropriate) prior to the initiation of any study
mandated procedures or assessments
2. A confirmed diagnosis of Pulmonary Hypertension Group 1 (PAH) who have either idiopathic PAH
(IPAH), heritable PAH, anorexigen-induced PAH, associated PAH (APAH) with connective tissue
disease (CTD), APAH with repaired simple congenital systemic to pulmonary shunt (i.e., atrial septal
defect [ASD], ventricular septal defect [VSD] and/or patent ductus arteriosus [PDA]; complete repair at
least 1 year prior to Screening) or APAH with human immunodeficiency virus (HIV)
3. Confirmation of PAH diagnosis at the time of Baseline RHC according to the following definition:
mPAP >= 25 mmHg at rest, with a concomitant mean pulmonary capillary wedge pressure (mPCWP),
mean left atrial pressure (mLAP), or left ventricular end diastolic pressure (LVEDP) <= 15 mmHg and a
PVR >= 240 dynes.sec/cm-5
4. 6MWD at least 100 meters and no greater than 450 meters
5. The subject is receiving at least one approved PAH therapy and is clinically symptomatic from PAH (e.g.,
onset or increased dyspnea on exertion, dizziness, near-syncope, syncope, chest pain or peripheral edema)
6. Background PAH medication doses (including calcium channel blockade if being used to treat PAH) must
be stable for at least 12 weeks prior to Screening
7. If on background conventional therapy (e.g., digoxin, diuretics, supplemental oxygen, anticoagulation), it
must have been started at least 30 days prior to Screening and be on a stable dose for at least 30 days
except for anticoagulation dose
8. If previously treated with an endothelin receptor antagonist (ERA), phosphodiesterase-5 (PDE-5)
inhibitor, prostacyclin or a prostacyclin analog and is no longer on said treatment at Screening (per
inclusion criteria as above), subject must have been off said treatment for > 90 days at Screening
9. If previously treated with a calcium channel blocker as treatment for PAH and is no longer on the calcium
channel blocker treatment at Screening (per inclusion criteria as above), subject must have been off the
calcium channel blocker treatment for > 90 days at Screening
10. Age between 16 and 80 years (inclusive)
11. Male height <= 200 cm (6[Single Quote]7
* ) or Female height <= 210 cm (6[Single Quote]11
* )
12. Subjects are willing and considered in the judgment of the Investigator able to use the INOpulse DS device continuously for up to 24 hours per day
13. Females of childbearing potential must have a negative pre-treatment serum pregnancy test and must beon a reliable method of contraception (including double protection if appropriate, e.g., for subjects concurrently treated with bosentan therapy)