Inhaled Treprostinil for Pulmonary Fibrosis

Recruiting in Palo Alto (17 mi)

+105 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: United Therapeutics

Must be taking: Nintedanib, Pirfenidone

Must not be taking: Prostacyclin, Endothelin antagonists

Disqualifiers: Pregnancy, IPF, Uncontrolled cardiac disease, others

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 2 Jurisdictions

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. ^{1 2 3 4 5}

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. ^{6 7 8 9 10}

Research Team

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.

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.

Trial 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 (Prostaglandin Analog)

Trial Overview The 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

Group I: Inhaled Treprostinil Experimental Treatment 2 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: Placebo Placebo Group 2 Interventions

Matching placebo inhaled using an ultrasonic nebulizer QID

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

Treprostinil (Remodulin) is a new treatment option for pulmonary hypertension, highlighting its potential as an effective therapy for this condition.

The study outlines a comprehensive multidisciplinary management plan that includes a flow chart to address high-risk issues associated with the use of treprostinil, emphasizing the importance of safety in its administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient safety challenges in treprostinil therapy. Roncesvalles, A., Lee, FW., Camamo, J., et al.[2017]

In a pilot study of 40 men who experienced incontinence after robot-assisted radical prostatectomy, the muscarinic receptor antagonist solifenacin was found to be well tolerated, with a 15% withdrawal rate due to side effects.

After 3 months of treatment, 53.8% of participants achieved continence (defined as using zero pads), suggesting potential efficacy, although it remains unclear if the improvement was directly due to the medication.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase 1 clinical trial of Vesicare™ (solifenacin) in the treatment of urinary incontinence after radical prostatectomy. Liss, MA., Morales, B., Skarecky, D., et al.[2018]

In a study of 94 patients with obstructive urination due to benign prostatic hyperplasia, the combination of finasteride and terazosin led to a significant reduction in prostate size by an average of 17% in 72% of patients over an average follow-up of 16.1 months.

Patients experienced a 55% increase in maximal urine flow rate, indicating improved urination, and the treatment was well tolerated, making it an effective conservative therapy option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[A trial of the use of finasteride and terazosin in patients with benign prostatic hyperplasia]. Pushkar', DIu., Kosko, DV., Loran, OB., et al.[2013]

References

1. England pubmed.ncbi.nlm.nih.gov

Tyvaso DPI: Drug-device characteristics and patient clinical considerations. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

An observational study of inhaled-treprostinil respiratory-related safety in patients with pulmonary arterial hypertension. [2022]

3. Switzerland pubmed.ncbi.nlm.nih.gov

A Comprehensive Review of Treprostinil Pharmacokinetics via Four Routes of Administration. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Patient safety challenges in treprostinil therapy. [2017]

5. United States pubmed.ncbi.nlm.nih.gov

Comparative bioavailability of inhaled treprostinil administered as LIQ861 and Tyvaso® in healthy subjects. [2021]

6. Germany pubmed.ncbi.nlm.nih.gov

Comparison of the antimuscarinic and antispasmodic actions of racemic oxybutynin and desethyloxybutynin and their enantiomers with those of racemic terodiline. [2017]

7. India pubmed.ncbi.nlm.nih.gov

Treating overactive bladder symptoms after transurethral prostatic surgery for benign prostatic hyperplasia - Which medication to choose? [2023]

8. Germany pubmed.ncbi.nlm.nih.gov

Efficacy, tolerability and safety profile of propiverine in the treatment of the overactive bladder (non-neurogenic and neurogenic). [2019]

9. United States pubmed.ncbi.nlm.nih.gov

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]