Inhaled Treprostinil for Chronic Obstructive Pulmonary Disease

(Tyvaso Trial)

Overseen By Savannah Barbieri

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Bastiaan Driehuys

Must be taking: Inhaled corticosteroids, Long-acting beta agonists

Disqualifiers: Asthma, Lung resection, Interstitial lung disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether inhaled Treprostinil can help individuals with chronic obstructive pulmonary disease (COPD) breathe better by improving oxygen delivery and blood flow in the lungs. Participants will inhale the medication four times daily for four weeks. The trial seeks individuals with a history of chronic bronchitis, who are current or former heavy smokers, and who require supplemental oxygen either at rest or during exercise. Researchers will use MRIs and breathing tests to assess the treatment's impact on lung function. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not require you to stop your current COPD medications. You need to be on stable maintenance medications, like inhaled corticosteroids or long-acting bronchodilators, without changes for the past 3 months.

Is there any evidence suggesting that inhaled Treprostinil is likely to be safe for humans?

Research has shown that inhaled Treprostinil could be a safe treatment for certain lung conditions. In studies, most patients did not experience major problems with their oxygen levels while using this medication, suggesting it might be well-tolerated.

Although some sources indicate that the safety of inhaled Treprostinil isn't completely clear, it is often considered a promising option for individuals with lung issues such as pulmonary hypertension and COPD. However, its effectiveness and the balance of risks and benefits can vary, particularly for conditions like COPD.

Overall, the treatment appears safe, but individual experiences may differ. Researchers closely monitor trial participants for any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for COPD?

Inhaled Treprostinil is unique because it offers a new delivery method for treating Chronic Obstructive Pulmonary Disease (COPD) with hypoxemia. Unlike standard treatments, which often involve oral medications or nebulizers, this inhaled formulation allows for direct delivery to the lungs, potentially enhancing the drug’s effectiveness and reducing systemic side effects. Researchers are excited about this approach because it targets the pulmonary system directly, which could improve oxygenation and exercise capacity for patients with preserved DLCO. This precise targeting may lead to better outcomes and a higher quality of life for COPD patients.

What evidence suggests that inhaled Treprostinil might be an effective treatment for COPD?

Research has shown that inhaled Treprostinil effectively treats certain lung conditions, such as pulmonary hypertension. It has increased the distance patients with interstitial lung disease can walk in six minutes. Although it did not improve walking distance for those with pulmonary hypertension and COPD, the treatment has shown benefits in similar lung issues by enhancing exercise ability. Inhaled Treprostinil relaxes the blood vessels in the lungs, improving blood flow and oxygen delivery. These effects suggest it could help treat COPD, particularly by improving lung function. Participants in this trial will receive inhaled Treprostinil to evaluate its potential benefits for COPD patients with hypoxemia and preserved DLCO. ^{2 4 5 6 7}

Are You a Good Fit for This Trial?

Adults aged 40 or older with COPD and low oxygen levels who can still get enough oxygen into their blood (DLCO ≥45%) may join this trial. They'll use a Tyvaso nebulizer to inhale Treprostinil, aiming to improve lung blood flow and oxygen delivery.

Inclusion Criteria

Willing and able to give informed consent and adhere to visit and protocol schedules (consent must be obtained prior to any study procedures)

I am 40 years old or older.

I have smoked 20 or more pack-years.

Exclusion Criteria

History of asthma

I have had surgery to remove part of my lung or to peel off the lining.

Positive pregnancy test for women of childbearing potential

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants use the Tyvaso nebulizer (inhaled Treprostinil) 4 times daily for 4 weeks, starting at 3 breaths per session and increasing to a maximum of 6 breaths per session as tolerated.

4 weeks

Baseline and end of treatment visits (in-person), phone check-in midway

Follow-up

Participants are monitored for safety and effectiveness after treatment, including adverse event monitoring and assessment of lung function.

4 weeks

Phone check-in after treatment

What Are the Treatments Tested in This Trial?

Interventions

• Inhaled Treprostinil

Trial Overview The study tests if inhaled Treprostinil from a Tyvaso nebulizer increases lung capillary blood volume and improves oxygen delivery without significantly affecting pulmonary vascular resistance. It includes MRI scans, walking tests, and echocardiograms before and after treatment.

How Is the Trial Designed?

Group I: Inhaled Treprostinil in COPD Patients With Hypoxemia and Preserved DLCO Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Bastiaan Driehuys

Lead Sponsor

Trials

15

Recruited

1,100+

Yuh Chin T Huang, MD, MHS

Lead Sponsor

United Therapeutics

Industry 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

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38811045/

Inhaled treprostinil in pulmonary hypertension associated ... The PERFECT study (ClinicalTrials.gov: NCT03496623) evaluated the safety and efficacy of inhaled treprostinil (iTRE) in this patient population.

2. publications.ersnet.org publications.ersnet.org/content/erj/63/6/2400172

Inhaled treprostinil in pulmonary hypertension associated with ... In addition, patients treated with iTRE showed no improvement in 6MWD when compared with placebo. Overall, this study showed that the risks in treating PH-COPD ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03496623

NCT03496623 | A Phase 3 Adaptive Study to Evaluate the ... The primary objective of this study is to demonstrate the efficacy of inhaled treprostinil compared to placebo in improving exercise ability as measured by ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S095461112400310X

Efficacy and determinants of response to inhaled ... Treatment with inhaled treprostinil for six months increased the 6MWT by more than 30 m in 38.5 % of PH-ILD patients. •. Lower RV tissue Doppler S′ velocity and ...

5. nejm.org nejm.org/doi/full/10.1056/NEJMoa2008470

Inhaled Treprostinil in Pulmonary Hypertension Due to ... In patients with pulmonary hypertension due to interstitial lung disease, inhaled treprostinil improved exercise capacity from baseline.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5448530/

The safety and tolerability of inhaled treprostinil in patients ... The small number of patients limits firm conclusions; however, inhaled treprostinil did not seem to adversely impact oxygenation in the majority of the study ...

7. journal.chestnet.org journal.chestnet.org/article/S0012-3692(22)03339-6/fulltext

USE OF INHALED TREPROSTINIL IN PULMONARY ... Inhaled Treprostinil can be a potentially safe and effective option for patients with pre-capillary PH and COPD.