20 Participants Needed

Riociguat + Treprostinil for Pulmonary Arterial Hypertension

VB
Overseen ByValerie Boss, MS
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: University of Arizona
Must be taking: Treprostinil
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
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

Trial Summary

What is the purpose of this trial?

The purpose of this study is to determine if there is a greater effect to patients with advanced pulmonary arterial hypertension (PAH) by using a combination of two drugs, Treprostinil and Riociquat versus Treprostinil alone

Will I have to stop taking my current medications?

The trial requires that you have not taken any pulmonary vasoactive medication in the last 12 weeks and that you are not on certain medications like strong CYP3A4 inhibitors or nitrates. It's best to discuss your current medications with the trial team to see if any need to be stopped.

What data supports the effectiveness of the drug combination of Riociguat and Treprostinil for treating pulmonary arterial hypertension?

Research shows that Treprostinil, when used alone or with other drugs, helps improve symptoms in people with pulmonary arterial hypertension. It has been effective in various forms, like subcutaneous (under the skin) and oral, for improving heart and lung function in these patients.12345

Is the combination of Riociguat and Treprostinil safe for humans?

Riociguat and Treprostinil have been studied separately and in combination for conditions like pulmonary hypertension. Riociguat is generally well-tolerated, but can cause low blood pressure, and Treprostinil can cause pain at the injection site. The combination has shown positive outcomes in some cases, but more research is needed to fully understand its safety.24678

How is the drug Riociguat + Treprostinil unique for treating pulmonary arterial hypertension?

The combination of Riociguat and Treprostinil is unique because Riociguat directly stimulates the production of cyclic GMP, which helps relax blood vessels in the lungs, while Treprostinil is a long-acting prostacyclin analog that improves blood flow. This combination may offer enhanced treatment outcomes for patients with pulmonary arterial hypertension compared to using each drug alone.578910

Research Team

FR

Franz Rischard, DO

Principal Investigator

University of Arizona

Eligibility Criteria

This trial is for individuals with advanced pulmonary arterial hypertension (PAH), specifically WHO Category I, showing certain heart pressure levels during tests. Participants must require Treprostinil as determined by a pulmonary hypertension specialist.

Inclusion Criteria

Resting mPAP ≥ 25 mmHg with a wedge pressure of ≤ 15mmHg during right heart catheterization.
My doctor says I need injections for my lung condition.
I have been diagnosed with a specific type of high blood pressure in the lungs.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a combination of Treprostinil and Riociguat or Treprostinil alone to evaluate effects on right ventriculo-vascular coupling and morphology

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Riociguat Pill
  • Treprostinil Injectable Product
Trial OverviewThe study aims to compare the effects of using both Treprostinil (an injectable drug) and Riociguat (a pill) versus just Treprostinil alone on heart function and shape in patients with advanced PAH.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: ExperimentalExperimental Treatment2 Interventions
Treprostinil and Riociguat

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Arizona

Lead Sponsor

Trials
545
Recruited
161,000+

Findings from Research

In a study of 32 adult patients with congenital heart disease-associated pulmonary arterial hypertension (CHD-PAH), subcutaneous treprostinil treatment for 12 months resulted in a significant increase in exercise capacity, as measured by a 114-meter improvement in the 6-minute walk distance (6-MWD) (P<0.001).
The treatment was generally safe, with the most common side effects being infusion-site erythema and pain; however, one patient discontinued due to intolerable pain, and no major complications were reported.
Subcutaneous treprostinil in congenital heart disease-related pulmonary arterial hypertension.Skoro-Sajer, N., Gerges, C., Balint, OH., et al.[2019]
The combination of oral sildenafil and inhaled treprostinil was found to be safe and well tolerated in patients with pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH), showing no relevant side effects during the study.
This combination therapy resulted in significant improvements in pulmonary vascular resistance and mean pulmonary arterial pressure, indicating a strong potential for effective long-term treatment of these conditions.
Acute effects of the combination of sildenafil and inhaled treprostinil on haemodynamics and gas exchange in pulmonary hypertension.Voswinckel, R., Reichenberger, F., Enke, B., et al.[2022]
In a study of 8 young children with refractory pulmonary arterial hypertension, subcutaneous treprostinil therapy led to significant early improvements in functional class and hemodynamics, with 7 out of 8 patients showing positive responses.
The treatment was generally well-tolerated, with manageable site pain in all but one child, suggesting that treprostinil could be a valuable rescue therapy for this condition, although more research is needed with larger patient groups.
Add-on therapy with subcutaneous treprostinil for refractory pediatric pulmonary hypertension.Levy, M., Celermajer, DS., Bourges-Petit, E., et al.[2017]

References

Subcutaneous treprostinil in congenital heart disease-related pulmonary arterial hypertension. [2019]
Acute effects of the combination of sildenafil and inhaled treprostinil on haemodynamics and gas exchange in pulmonary hypertension. [2022]
Add-on therapy with subcutaneous treprostinil for refractory pediatric pulmonary hypertension. [2017]
Safety, Tolerability and Clinical Effects of a Rapid Dose Titration of Subcutaneous Treprostinil Therapy in Pulmonary Arterial Hypertension: A Prospective Multi-Centre Trial. [2022]
Oral treprostinil for the treatment of pulmonary arterial hypertension in patients on background endothelin receptor antagonist and/or phosphodiesterase type 5 inhibitor therapy (the FREEDOM-C study): a randomized controlled trial. [2022]
Riociguat: a novel new drug for treatment of pulmonary hypertension. [2017]
Combination therapy with riociquat and inhaled treprostinil in inoperable and progressive chronic thromboembolic pulmonary hypertension. [2020]
Riociguat treatment in patients with chronic thromboembolic pulmonary hypertension: Final safety data from the EXPERT registry. [2021]
Treprostinil-based therapy in the treatment of moderate-to-severe pulmonary arterial hypertension: long-term efficacy and combination with bosentan. [2018]
10.United Statespubmed.ncbi.nlm.nih.gov
Delivery of intravenous treprostinil at low infusion rates using a miniaturized infusion pump in patients with pulmonary arterial hypertension. [2019]