698 Participants Needed

Inhaled Treprostinil for Pulmonary Fibrosis

Recruiting at 110 trial locations
UT
AM
DB
Overseen ByDebabrata Bandyopadhyay, MD
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: United Therapeutics
Must be taking: Nintedanib, Pirfenidone
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

Study RIN-PF-305 is designed to evaluate the safety and efficacy of inhaled treprostinil in subjects with progressive pulmonary fibrosis (PPF) over a 52-week period.

Do I need to stop my current medications to join the trial?

The trial does not require you to stop your current medications. If you are on nintedanib or pirfenidone, you should continue them if you have been taking them for at least 90 days. If you are on immunosuppressive agents, you should have been on them for at least 120 days before starting the trial.

Is inhaled treprostinil safe for humans?

Inhaled treprostinil, used for conditions like pulmonary arterial hypertension, has been shown to be generally safe in humans. Common side effects include cough, throat irritation, and nasal discomfort, but it is well tolerated overall.12345

How does the drug Inhaled Treprostinil differ from other treatments for pulmonary fibrosis?

Inhaled Treprostinil is unique because it is administered through inhalation, directly targeting the lungs, which may enhance its effectiveness in treating pulmonary fibrosis. This method of delivery is different from many other treatments that are taken orally or intravenously.678910

Eligibility Criteria

Adults diagnosed with progressive pulmonary fibrosis, who are not pregnant or breastfeeding, can join this trial. They must use contraception if applicable and have been on certain treatments for at least 120 days. People with recent lung infections, high oxygen needs, a specific type of lung scarring called IPF, or those using certain PAH therapies recently cannot participate.

Inclusion Criteria

My lung scans show more than 10% fibrosis.
I will use a condom during treatment and for 48 hours after stopping the study drug.
I am not taking nintedanib and pirfenidone at the same time.
See 8 more

Exclusion Criteria

I have severe lung obstruction or more damage from emphysema than scarring on a lung scan.
I haven't taken part in any medical trials involving treatments in the last 30 days.
I have taken medication for pulmonary arterial hypertension in the last 60 days.
See 9 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive inhaled treprostinil or placebo for 52 weeks, with dose titration as tolerated

52 weeks
6 visits (in-person) at Weeks 4, 8, 16, 28, 40, and 52

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term after completing the main study

Treatment Details

Interventions

  • Inhaled Treprostinil
Trial OverviewThe study is testing the safety and effectiveness of inhaled treprostinil delivered via an ultrasonic nebulizer compared to a placebo over one year. It aims to see if this treatment can help people with worsening lung scarring despite standard care.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Inhaled TreprostinilExperimental Treatment2 Interventions
Treprostinil for inhalation solution (0.6 mg/mL) delivered via an ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath. Inhaled QID and titrated up to a target of 12 breaths QID or until the subject reaches their maximum clinically tolerated dose.
Group II: PlaceboPlacebo Group2 Interventions
Matching placebo inhaled using an ultrasonic nebulizer QID

Inhaled Treprostinil is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Tyvaso for:
  • Pulmonary arterial hypertension (PAH)
  • Pulmonary hypertension associated with interstitial lung disease (PH-ILD)
🇪🇺
Approved in European Union as Tyvaso for:
  • Pulmonary arterial hypertension (PAH)
  • Pulmonary hypertension associated with interstitial lung disease (PH-ILD)

Find a Clinic Near You

Who Is Running the Clinical Trial?

United Therapeutics

Lead Sponsor

Trials
112
Recruited
14,500+

Dr. Martine Rothblatt

United Therapeutics

Chief Executive Officer since 1996

PhD in Medical Ethics from the Royal London College of Medicine and Dentistry, JD and MBA from UCLA

Dr. Michael Benkowitz

United Therapeutics

Chief Medical Officer since 2023

MD from Harvard Medical School

Findings from Research

Tyvaso DPI is a portable, breath-powered inhaler that delivers treprostinil for treating pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease, offering a passive administration method that enhances patient compliance.
In clinical studies, Tyvaso DPI demonstrated similar drug exposure and safety profiles to nebulized treprostinil, with a low incidence of adverse events and high patient satisfaction, making it a suitable option for patients seeking a convenient and effective treatment.
Tyvaso DPI: Drug-device characteristics and patient clinical considerations.McEvoy, C., Argula, R., Sahay, S., et al.[2023]
Inhaled treprostinil (Tyvaso) is a safe and effective treatment for pulmonary arterial hypertension (PAH), with a large study involving 1,333 patients showing it can be well tolerated in routine clinical care.
While patients treated with inhaled treprostinil reported a higher incidence of respiratory-related adverse events, such as cough and throat irritation, these events were consistent with the known safety profile of the drug.
An observational study of inhaled-treprostinil respiratory-related safety in patients with pulmonary arterial hypertension.Zamanian, RT., Levine, DJ., Bourge, RC., et al.[2022]
Treprostinil is an effective treatment for pulmonary arterial hypertension (PAH) and is available in three formulations: intravenous/subcutaneous (Remodulin®), inhaled (Tyvaso®), and oral (Orenitram®).
Pharmacokinetic studies in both healthy volunteers and PAH patients provide important insights that can help clinicians make informed decisions about the most suitable route of administration for individual patients.
A Comprehensive Review of Treprostinil Pharmacokinetics via Four Routes of Administration.Kumar, P., Thudium, E., Laliberte, K., et al.[2018]

References

Tyvaso DPI: Drug-device characteristics and patient clinical considerations. [2023]
An observational study of inhaled-treprostinil respiratory-related safety in patients with pulmonary arterial hypertension. [2022]
A Comprehensive Review of Treprostinil Pharmacokinetics via Four Routes of Administration. [2018]
Patient safety challenges in treprostinil therapy. [2017]
Comparative bioavailability of inhaled treprostinil administered as LIQ861 and Tyvaso® in healthy subjects. [2021]
Comparison of the antimuscarinic and antispasmodic actions of racemic oxybutynin and desethyloxybutynin and their enantiomers with those of racemic terodiline. [2017]
Treating overactive bladder symptoms after transurethral prostatic surgery for benign prostatic hyperplasia - Which medication to choose? [2023]
Efficacy, tolerability and safety profile of propiverine in the treatment of the overactive bladder (non-neurogenic and neurogenic). [2019]
Phase 1 clinical trial of Vesicare™ (solifenacin) in the treatment of urinary incontinence after radical prostatectomy. [2018]
10.Russia (Federation)pubmed.ncbi.nlm.nih.gov
[A trial of the use of finasteride and terazosin in patients with benign prostatic hyperplasia]. [2013]