A Randomized, Double-blind, Placebo-controlled, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis (TETON-1)

Plain English Summary

Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis is a Phase 3 clinical trial sponsored by United Therapeutics studying Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease. Tests the effectiveness of inhaled treprostinil compared to placebo in slowing lung function decline in IPF patients. For adults aged 40+ with confirmed IPF diagnosis and stable lung function. Participation involves daily inhaler use and regular clinic visits over 52 weeks. Alternative treatments include oral medications like pirfenidone and nintedanib. The trial aims to enroll 598 participants.

Official Summary

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.

Who Can Participate

Here is what you need to know about eligibility for this trial. Eligible if over 40, diagnosed with IPF, and has stable lung function. Not eligible if pregnant, has severe lung obstruction, or is using other investigational drugs. Age: 40+ years Health: Confirmed IPF diagnosis, stable lung function, no severe cardiac issues This trial is studying Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures the change in lung function, helping patients understand if their condition is improving or worsening. The specific primary outcome measures are: Change in Absolute FVC from Baseline to Week 52 (Baseline to Week 52). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial aims to fill a gap in treatment options for IPF by evaluating a new inhaled therapy. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease, where improved treatment options are needed.

Investor Insight

Market size is significant, with a competitive landscape and a high probability of approval given the unmet need in IPF treatment. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage. The large enrollment target of 598 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor about your lung function and IPF diagnosis. Daily inhaler use and regular clinic visits are required. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 598 participants

Interventions

• DRUG: Placebo — Placebo administered QID
• DRUG: Inhaled Treprostinil — Inhaled treprostinil (6 mcg/breath) administered QID
• DEVICE: Treprostinil Ultrasonic Nebulizer — Treprostinil ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath.

Primary Outcomes

• Change in Absolute FVC from Baseline to Week 52 (Baseline to Week 52)

Secondary Outcomes

• Time to Clinical Worsening (Baseline to Week 52)
• Time to First Acute Exacerbation of IPF (Baseline to Week 52)
• Overall Survival at Week 52 (Week 52)
• Change in % Predicted FVC from Baseline to Week 52 (Baseline to Week 52)
• Change in K-BILD Questionnaire Score from Baseline to Week 52 (Baseline to Week 52)

Full Eligibility Criteria

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 permitted.
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 cooperation.
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.

Trial Locations

• The University of Alabama at Birmingham, Birmingham, Alabama, United States
• Banner University Medical Center-Phoenix, Phoenix, Arizona, United States
• Arizona Pulmonary Specialists, Ltd., Phoenix, Arizona, United States
• St. Joseph's Hospital and Medical Center - Norton Thoracic Institute, Phoenix, Arizona, United States
• University of Arizona, Tucson, Arizona, United States
• UC San Diego Health, La Jolla, California, United States
• David Geffen School of Medicine at UCLA, Los Angeles, California, United States
• NewportNativeMD, Inc, Newport Beach, California, United States
• University of California, Irvine, Orange, California, United States
• Palmtree Clinical Research, Inc., Palm Springs, California, United States
• ...and 10 more locations

Frequently Asked Questions

Related Conditions

• Idiopathic Pulmonary Fibrosis
• Interstitial Lung Disease
• Chronic Obstructive Pulmonary Disease (COPD)
• Pulmonary Hypertension
• Sarcoidosis
• Lupus
• Asthma
• Cystic Fibrosis
• Bronchiectasis
• Pulmonary Embolism

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