Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis


Trial ID: NCT04708782


Study RIN-PF-301 is designed to evaluate the superiority of inhaled treprostinil against placebo for the change in absolute forced vital capacity (FVC) from baseline to Week 52.

Official Title

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis

Stanford Investigator(s)

Rishi Raj
Rishi Raj

Clinical Professor, Medicine - Pulmonary, Allergy & Critical Care Medicine


Inclusion Criteria:

   1. Subject gives voluntary informed consent to participate in the study.

   2. Subject is ≥40 years of age, inclusive, at the time of signing informed consent.

   3. The subject has a diagnosis of IPF based on the 2018 ATS/ERS/JRS/ALAT Clinical
   Practice Guideline (Raghu 2018) and confirmed by central review of high-resolution
   computed tomography (HRCT) (performed within the previous 12 months), and if
   available, surgical lung biopsy.

   4. FVC ≥45% predicted at Screening.

   5. Subjects on pirfenidone or nintedanib must be on a stable and optimized dose for ≥30
   days prior to Baseline. Concomitant use of both pirfenidone and nintedanib is not

   6. Women of childbearing potential must be non-pregnant (as confirmed by a urine
   pregnancy test at Screening and Baseline) and non-lactating, and will abstain from
   intercourse (when it is in line with their preferred and usual lifestyle) or use 2
   medically acceptable, highly effective forms of contraception for the duration of the
   study, and at least 30 days after discontinuing study drug.

   7. Males with a partner of childbearing potential must use a condom for the duration of
   treatment and for at least 48 hours after discontinuing study drug.

   8. In the opinion of the Investigator, the subject is able to communicate effectively
   with study personnel, and is considered reliable, willing, and likely to be
   cooperative with protocol requirements, including attending all study visits.

Exclusion Criteria:

   1. Subject is pregnant or lactating.

   2. Subject has primary obstructive airway physiology: FEV1/FVC <0.70 at Screening.

   3. The subject has shown intolerance or significant lack of efficacy to a prostacyclin or
   prostacyclin analogue that resulted in discontinuation or inability to effectively
   titrate that therapy.

   4. The subject has received any PAH-approved therapy, including prostacyclin therapy
   (epoprostenol, treprostinil, iloprost, or beraprost; except for acute vasoreactivity
   testing), IP receptor agonists (selexipag), endothelin receptor antagonists,
   phosphodiesterase type 5 inhibitors (PDE5-Is), or soluble guanylate cyclase
   stimulators within 60 days prior to Baseline. As needed use of a PDE5-I for erectile
   dysfunction is permitted, provided no doses are taken within 48 hours of any
   study-related efficacy assessments.

   5. Use of any of the following medications: azathioprine (AZA), cyclosporine,
   mycophenolate mofetil, tacrolimus, oral corticosteroids (OCS) >20 mg/day or the
   combination of OCS+AZA+N-acetylcysteine within 30 days prior to Baseline;
   cyclophosphamide within 60 days prior to Baseline; or rituximab within 6 months prior
   to Baseline.

   6. The subject is receiving >10 L/min of oxygen supplementation by any mode of delivery
   at rest at Baseline.

   7. Exacerbation of IPF or active pulmonary or upper respiratory infection within 30 days
   prior to Baseline. Subjects must have completed any antibiotic or steroid regimens for
   treatment of the infection or acute exacerbation more than 30 days prior to Baseline
   to be eligible. If hospitalized for an acute exacerbation of IPF or a pulmonary or
   upper respiratory infection, subjects must have been discharged more than 90 days
   prior to Baseline to be eligible.

   8. Uncontrolled cardiac disease, defined as myocardial infarction within 6 months prior
   to Baseline or unstable angina within 30 days prior to Baseline.

   9. In the opinion of the Investigator, the subject has any condition that would interfere
   with the interpretation of study assessments or would impair study participation or

10. Use of any other investigational drug/device or participation in any investigational
   study in which the subject received a medical intervention (ie, procedure, device,
   medication/supplement) within 30 days prior to Screening. Subjects participating in
   non-interventional, observational, or registry studies are eligible.

11. Life expectancy <6 months due to IPF or a concomitant illness.

12. Acute pulmonary embolism within 90 days prior to Baseline.


drug: Placebo

drug: Inhaled Treprostinil

device: Treprostinil Ultrasonic Nebulizer


Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305