Epoprostenol for Patients on ECMO

(PACE Trial)

AH
GK
Overseen ByGarrett Keim, MD, MSCE
Age: < 18
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Garrett Keim
Must be taking: Unfractionated Heparin, Bivalirudin
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called epoprostenol (Veletri) to determine if it can safely prevent blood clots in children on ECMO. ECMO supports the heart and lungs when they are not functioning well. The trial aims to identify the safest dose of epoprostenol and assess its impact on blood clotting in children who are stable on ECMO. It suits children on ECMO with stable blood pressure who are already receiving blood thinners. As a Phase 1, Phase 2 trial, it focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that epoprostenol, also known as Veletri, is generally safe for patients. One study found no new safety issues, and most patients tolerated it well. This medication primarily treats high blood pressure in the lungs and has FDA approval for this use. However, it can affect blood clotting, which is crucial for people on ECMO (a machine that aids breathing and circulation) because the machine can cause blood clots. The current study aims to determine the safest dose for people on ECMO and closely monitors side effects such as changes in blood pressure, unexpected bleeding, and fluid in the lungs.12345

Why do researchers think this study treatment might be promising?

Epoprostenol (Veletri) is unique because it directly targets blood vessels to cause dilation, potentially improving blood flow in patients on ECMO (extracorporeal membrane oxygenation) support. Unlike standard treatments for ECMO patients, which often focus on mechanical support and anticoagulation, epoprostenol works by mimicking a natural compound in the body called prostacyclin. Researchers are excited about epoprostenol because it might enhance oxygen delivery and reduce complications associated with ECMO therapy more effectively than current options. This targeted approach could lead to better outcomes and faster recovery times for patients.

What evidence suggests that epoprostenol might be an effective treatment for ECMO patients?

Research has shown that epoprostenol, a medication often used for high blood pressure in the lungs, can help prevent blood clots by reducing platelet activity. This is crucial for patients using ECMO, a machine that supports heart and lung function, where clotting can be problematic. Studies have found that epoprostenol can improve oxygen levels in the blood for patients with serious lung issues. In this trial, participants will receive different doses of epoprostenol based on their weight, potentially lowering the risk of clotting in ECMO situations due to its ability to reduce platelet activity.678910

Are You a Good Fit for This Trial?

This trial is for children on ECMO (a heart-lung support machine) who have had stable blood pressure for at least 12 hours and have been safely on blood thinners for at least 6 hours. Participants must be able to give consent or have a guardian provide it.

Inclusion Criteria

Written informed consent provided by the patient's parent or Legally Authorized Representative (LAR)
I am 17 years old or younger.
I was at least 37 weeks gestational age when ECMO started.
See 2 more

Exclusion Criteria

Positive pregnancy test
Severe coagulopathy with platelet count < 50,000/ uL or fibrinogen < 50 mg/dL at baseline
Known hypersensitivity to epoprostenol
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a 48-hour infusion of epoprostenol with associated blood sampling and adverse event monitoring

48 hours
Continuous monitoring during infusion

Follow-up

Participants are monitored for safety and effectiveness after treatment, including adverse hemodynamic effects, bleeding, and pulmonary edema

72 hours
Monitoring within 72 hours post-infusion

Long-term Follow-up

Observation of circuit integrity and anticoagulation dose adjustments during ECMO duration

ECMO Duration

What Are the Treatments Tested in This Trial?

Interventions

  • Epoprostenol (Veletri)

Trial Overview

The study tests different doses of epoprostenol, given through the ECMO circuit as an extra blood thinner, to find the safest dose and see how it affects platelets and clotting. Each child receives one dose over 48 hours with close monitoring.

How Is the Trial Designed?

6

Treatment groups

Experimental Treatment

Group I: Statum B: 8 ng/kg/minExperimental Treatment1 Intervention
Group II: Statum B: 4 ng/kg/minExperimental Treatment1 Intervention
Group III: Statum B: 12 ng/kg/minExperimental Treatment1 Intervention
Group IV: Statum A: 8 ng/kg/minExperimental Treatment1 Intervention
Group V: Statum A: 4 ng/kg/minExperimental Treatment1 Intervention
Group VI: Statum A: 12 ng/kg/minExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Garrett Keim

Lead Sponsor

Citations

Epoprostenol Delivered via High Flow Nasal Cannula for ...

After inhaling epoprostenol, subjects' SpO2/FIO2 ratio improved from 107.5 ± 26.3 to 125.5 ± 31.6. Five subjects (45.5%) had SpO2/FIO2 ...

2.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/39456115/

VENO-VENOUS EXTRACORPOREAL MEMBRANE ...

VV ECMO patients had higher survival rates at discharge when compared to the matched conventional treatment group (70% vs. 41%, P < 0.001).

Extracorporeal Membrane Oxygenation for the Treatment ...

VA-ECMO provides essential cardiopulmonary support, although data outcomes remain limited. We sought to describe the characteristics and ...

Predictors of mortality following extracorporeal membrane ...

Overall mortality was ~66% at median 28 days follow-up and significantly depended upon pH < 7.3 (HR = 3.56; p < 0.001), and age ≥ 65 years p = 0.001).

Outcomes of Extracorporeal Membrane Oxygenation in ... - PMC

Survival to hospital discharge was 62.3% Kon and colleagues17 have reported 100% survival after decannulation of 13 patients with COVID-19.

Conversion from Veno-Venous to Veno-Arterial or Hybrid ...

Ethical Approval: The ELSO Scientific Oversight Committee (SOC) reviewed and validated the current data request and the study protocol. Keywords ...

Inhaled Epoprostenol Compared With Nitric Oxide for Right ...

One of these patients died within 30 days, was supported preoperatively with venoarterial extracorporeal membrane oxygenation, and required an aortic valve ...

Tolerability, safety and survival in patients with severe ...

The study shows that epoprostenol (Veletri®) treatment did not reveal new safety or tolerability aspects and was feasible in most patients. The survival rate ...

(PDF) The use of extracorporeal membrane oxygenation in ...

CONCLUSIONS: In patients with post-AMI MC, VA-ECMO provides haemodynamic stabilizations and carries a potential to reverse other- wise lethal course. ECMO ...

Clinical Policy: Epoprostenol (Flolan, Veletri)

Epoprostenol (Flolan®, Veletri®) is a prostacyclin. FDA Approved Indication(s). Flolan and Veletri are indicated for the treatment of pulmonary ...