Inhaled Treprostinil for Pulmonary Hypertension

Overseen By Colleen Irlbeck

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Mayo Clinic

Disqualifiers: Pregnancy, Inability to exercise, 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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 3 months

29 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing an inhaled medication called treprostinil on patients with a lung condition that makes it hard for them to exercise. The medication helps by improving blood flow in the lungs. Researchers want to see if it makes exercise easier and improves quality of life after a period of use. Treprostinil has been shown to improve exercise capacity and is more convenient due to its longer-lasting effects.

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

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Treprostinil for pulmonary hypertension?

Inhaled Treprostinil, such as Tyvaso DPI, is effective for treating pulmonary hypertension by improving walk distance in patients with symptoms. It is a patient-preferred option due to its ease of use and portability, and it has shown similar effectiveness and patient satisfaction compared to other forms of Treprostinil. ^{1 2 3 4 5}

Is inhaled Treprostinil safe for humans?

There is no relevant safety information about inhaled Treprostinil in the provided research articles. ^{6 7 8 9 10}

What makes the drug Tyvaso DPI unique for treating pulmonary hypertension?

Tyvaso DPI is unique because it is a portable, breath-powered inhaler that delivers treprostinil as a dry powder, making it easier to use compared to nebulizers. It targets the peripheral lungs effectively with minimal drug loss and is preferred by patients for its convenience and non-invasive nature. ^{1 2 3 4 5}

Research Team

Yogesh N Reddy, M.B.B.S

Principal Investigator

Mayo Clinic

Eligibility Criteria

Adults over 18 with suspected PAH-ILD undergoing right heart catheterization, and healthy volunteers without lung disease or pulmonary hypertension. It's not for anyone under 18.

Inclusion Criteria

Undergoing cardiac catheterization for clinical indications at Mayo Clinic Rochester over a 12-month period.

I am suspected to have high blood pressure in the lungs due to lung disease and will undergo a heart procedure.

I am healthy with no lung, heart, muscle diseases or high blood pressure in the lungs.

Exclusion Criteria

I am under 18 years old.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive inhaled treprostinil and undergo RHC while exercising

3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Treprostinil

Trial Overview The study is testing the drug treprostinil inhaled by patients with PAH-ILD to see how it affects their ability to exercise and quality of life after three months. Some may undergo optional muscle biopsies or RHC while exercising.

Participant Groups

Group I: PAH-ILD Patients Experimental Treatment 3 Interventions

Patients diagnosed with PAH-ILD will undergo RHC while exercising, receive inhaled treprostinil, and undergo an optional muscle biopsy.

Group II: Healthy Volunteers Experimental Treatment 2 Interventions

Healthy volunteers will undergo RHC while exercising and an optional muscle biopsy.

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

Approved in United States as Remodulin for:

• Pulmonary Arterial Hypertension

Approved in European Union as Remodulin for:

• Pulmonary Arterial Hypertension

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

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

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]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tyvaso DPI: Drug-device characteristics and patient clinical considerations. McEvoy, C., Argula, R., Sahay, S., et al.[2023]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Comprehensive Review of Treprostinil Pharmacokinetics via Four Routes of Administration. Kumar, P., Thudium, E., Laliberte, K., et al.[2018]

LIQ861, a dry-powder formulation of treprostinil, shows comparable bioavailability to the nebulized Tyvaso® inhalation solution, with exposure parameters indicating similar effectiveness in delivering the medication to patients with pulmonary arterial hypertension (PAH).

Both LIQ861 and Tyvaso® were well tolerated by participants, suggesting that LIQ861 offers a safe and convenient alternative for PAH patients, allowing for effective treatment in just 1 to 2 breaths.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative bioavailability of inhaled treprostinil administered as LIQ861 and Tyvaso® in healthy subjects. Roscigno, RF., Vaughn, T., Parsley, E., et al.[2021]

In a study of 57 men undergoing TUR-P or TUI-P, both solifenacin and mirabegron effectively reduced overactive bladder (OAB) symptoms at 2 weeks post-surgery, indicating their efficacy in managing immediate post-operative OAB symptoms.

Mirabegron demonstrated a better safety profile compared to solifenacin, as two patients in the solifenacin group experienced urinary retention, suggesting that β-3 agonists may be a safer option for patients after prostate surgery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treating overactive bladder symptoms after transurethral prostatic surgery for benign prostatic hyperplasia - Which medication to choose? Lee, CL., Kuo, HC.[2023]

References

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

Patient safety challenges in treprostinil therapy. [2017]

2. England pubmed.ncbi.nlm.nih.gov

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

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

Inhaled treprostinil for the treatment of pulmonary arterial hypertension. [2017]

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

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

6. India pubmed.ncbi.nlm.nih.gov

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

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

Effect of terfenadine on urination. [2013]

8. England pubmed.ncbi.nlm.nih.gov

Betrixaban - the next direct factor Xa inhibitor? [2017]

9. England pubmed.ncbi.nlm.nih.gov

Basic and clinical aspects of antimuscarinic agents used to treat overactive bladder. [2019]

10. Germany pubmed.ncbi.nlm.nih.gov

Prophylaxis of seasonal allergic rhinitis with a new antihistaminic drug. [2013]