Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System

NCT06080074 | Recruiting Phase 2 DMC FDA Drug FDA Device

• 1 other identifier: 72467
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 5

Brief Summary

There are two primary goals of this multicenter clinical trial that combines an FDA device trial and a phase II drug trial in the same study cohort. These two goals are to:

• What is the safety and effectiveness of the Cardiohelp device for pediatric ECMO?
• Should the Cardiohelp device be FDA-cleared for children based on the results of the study?
• What are the optimal performance specifications of the Cardiohelp device in children? The main questions the blood thinner randomized trial seeks to answer are:
• Which blood thinner is more promising (i.e., more effective and safer) in children on the Cardiohelp device?
• How should a pivotal trial of heparin vs. bivalirudin be designed so it is the most informative and efficient to determine the best blood thinner? Children who are receiving the Cardiohelp device will be approached and consented to participate if interested. For the Cardiohelp device trial, participants will undergo a standardized data collection to estimate survival to 30 days and the prevalence of serious adverse events like stroke, bleeding, and hemolysis. For the blood thinner randomized trial, participants will be randomized 1:1 to blood thinner strategy to determine which blood thinner has the fewest bleeding and clotting complications. For the Cardiohelp single-arm trial, participant outcomes will be compared to performance goals (PG) derived from the ECMO literature. For the blood thinner randomized trial, the amount of bleeding and clotting will be measured. The study is funded by an R01 grant from the FDA's Office of Orphan Product Development (OOPD).

Conditions

Heart Failure; Cardiogenic Shock; Congenital Heart Disease; Cardiomyopathies

Keywords

blood thinner; ECMO; heart failure; bivalirudin; heparin; FDA regulation; pediatric medical devices; 510k clearance

Outcome Measures

Primary Outcomes (8)

• Survival to 30 days, recovery, ventricular assist device implant or transplant in the absence of severe symptomatic stroke (primary device effectiveness)

  Survival in the absence of severe symptomatic stroke to 30 days or decannulation for recovery, transplant or VAD implant. For patients with stroke definitely or probably related to ECMO, symptoms and severity will be determined at hospital discharge or 90 days, whichever is first.

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Symptomatic stroke (primary device safety)

  For patients with stroke definitely or probably related to ECMO, the presence of stroke symptoms and its severity will be determined at hospital discharge or 90 days, whichever is first.

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Severe hemolysis (primary device safety)

  Severe hemolysis as defined by the plasma-free hemoglobin level \>100 mg/dL confirmed on repeat testing and outside the first 72 hours that is probably or definitely related to the device.

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Circuit change due to thrombus (primary device safety)

  ECMO circuit change due primarily to thrombus that is probably or definitely related to the device.

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Circuit change due primarily to device malfunction (primary device safety)

  Circuit change due primarily to device malfunction that is probably or definitely related to the device.

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Renal injury/failure (primary device safety)

  renal injury/failure defined as three times the upper limit of normal or 3X baseline if the baseline is above the upper limit of normal.

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Circuit-change free survival primarily due to thrombus (primary safety endpoint for anticoagulation RCT)

  Circuit-change free survival where the circuit is change is primarily due to thrombus and is definitely or probably related to the anticoagulant (primary safety endpoint for anticoagulation RCT)

  30 days of ECMO support or ECMO explant, whichever is earlier.

• Bleeding

  Total red blood cell transfusion volume in cc/kg/day probably or definitely related to the anticoagulant.

  30 days of ECMO support or ECMO explant, whichever is earlier.

Secondary Outcomes (2)

• Incidence of all serious adverse events

  30 days

• NHLBI bleeding and clotting score

  through 30 days of ECMO support or ECMO explant, whichever is earlier.

Study Arms (2)

Cardiohelp device managed according to a standardized guideline using heparin anticoagulation

EXPERIMENTAL

Participants receiving the Cardiohelp device for severe heart failure will be managed according to a standardized treatment guideline (equipment, monitoring, etc.) where heparin is the primary blood thinner to estimate the safety and effectiveness of the device and blood thinner strategy for up to 30 days of support.

Device: Cardiohelp device (VA-ECMO); Drug: Heparin

Cardiohelp device managed according to a standardized guideline using bivalirudin anticoagulation

EXPERIMENTAL

Participants receiving the Cardiohelp device for severe heart failure will be managed according to a standardized treatment guideline (equipment, monitoring, etc.) where bivalirudin is the primary blood thinner to estimate the safety and effectiveness of the device and blood thinner strategy for up to 30 days of support.

Device: Cardiohelp device (VA-ECMO); Drug: Bivalirudin

Interventions

Cardiohelp device (VA-ECMO) DEVICE

The Cardiohelp system is a compact cardiopulmonary support system used off-label for ECMO for patients with cardiac and/or respiratory failure. It is currently FDA-cleared for \<6 hours of support for cardiopulmonary bypass.

Cardiohelp device managed according to a standardized guideline using bivalirudin anticoagulation; Cardiohelp device managed according to a standardized guideline using heparin anticoagulation

Heparin DRUG

Blood thinner that works by inactivating factor Xa that is administered as a continuous intravenous infusion.

Cardiohelp device managed according to a standardized guideline using heparin anticoagulation

Bivalirudin DRUG

Blood thinner that works by inhibiting thrombin directly that is administered as a continuous intravenous infusion.

Cardiohelp device managed according to a standardized guideline using bivalirudin anticoagulation

Eligibility Criteria

• Age: 0 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age 0 to 16 years of age (i.e., \<17 years)
• Body weight 3 to 80 kilograms
• VA-ECMO use for primary cardiac failure using the Cardiohelp system.
• First ECMO run during the current hospitalization

You may not qualify if:

• Gestationally-corrected age \<37 weeks
• Bleeding or coagulopathy that is a contraindication to anticoagulation
• Irreversible renal, hepatic or lung failure
• Stroke or uncertain neurological status within the past 30 days
• Severely malnourished
• Use of an ECMO system other than the Cardiohelp
• VV-ECMO or ECMO for primary respiratory failure
• Goals of patient to focus on comfort measures only.
• Failure to separate from cardiopulmonary bypass
• Allergy or contraindication to receiving UFH or bivalirudin as a primary anticoagulant on ECMO.
• Patients who are pregnant or breastfeeding.
• Unable to undergo randomization within 30 hours following ECMO cannulation (randomized cohort only)

Sponsors & Collaborators

• Stanford University: lead
• Boston Children's Hospital: collaborator
• Duke University: collaborator
• FDA Office of Orphan Products Development: collaborator

Study Sites (5)

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

RECRUITING

Cuimc/Nyph

New York, New York, 10032, United States

RECRUITING

Duke University Hospital

Durham, North Carolina, 27710, United States

RECRUITING

Children's Health Dallas

Dallas, Texas, 95235, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

MeSH Terms

Conditions

Heart Failure; Shock, Cardiogenic; Heart Defects, Congenital; Cardiomyopathies

Interventions

Heparin; bivalirudin

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases; Myocardial Infarction; Myocardial Ischemia; Vascular Diseases; Infarction; Ischemia; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Necrosis; Shock; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Glycosaminoglycans; Polysaccharides; Carbohydrates

Study Officials

• Lynn Sleeper, ScD

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

• Caroline Ozment, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Christopher SD Almond, MD, MPH

CONTACT

6507237913; calmond@stanford.edu

Selena Gonzales, MPH

CONTACT

650-723-7913; selena.gonzales@stanford.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Model Details: Participants receiving ECMO with the Cardiohelp Device for severe heart failure will managed/treated according to a standardized treatment and monitoring protocol that includes anticoagulation strategy, circuit change criteria, weaning guidelines, and recording of adverse events using standardized SAE definitions adapted from the ECMO-CENTRAL guidelines. While the Cardiohelp device trial is a single-arm cohort study, the FDA allowed us to randomize patients to blood thinner (heparin and bivalirudin). Both blood thinners are in common use in the US--providing the FDA with safety and effectiveness data on each of the two anticoagulants in at least \~25 participants. Please note: Because the data fields for clinicaltrials.gov make it easier to report more robust details for a randomized clinical trial, we have chosen to report the details of the RCT in this section, understanding that the FDA clearance study is running in the background as a single arm observational study.

Study Record Dates

• First Submitted: October 6, 2023
• First Posted: October 12, 2023
• Study Start: April 15, 2025
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2029
• Last Updated: January 29, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)