Study of Autologous Bone Marrow Derived Mononuclear Cells for Treatment of Ebstein Anomaly

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-30

Study Completion Date

2021-03-18

Brief Summary

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The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNS) collected from bone marrow (BM) and using an add-on intramyocardial delivery for individuals with Ebstein anomaly undergoing surgical intervention compared to the control group undergoing the same surgical procedure without cell delivery. This add-on procedure has the potential to foster a new strategy for individuals with congenital heart disease.This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) incidence and severity of adverse event and 2) monitor changes in cardiac structure and function.

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Detailed Description

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This study is an open label Phase I trial to determine the safety and feasibility of bone marrow-derived mononuclear cells to the right ventricle of subjects with Ebstein anomaly at the time of a planned surgical Ebstein repair compared to the control group undergoing the same planned surgical intervention without the cell delivery add-on procedure. Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial. Subject/Family will decide to participate in cell delivery or control arm. Once informed consent has been obtained, subjects will undergo identical evaluation (other than Hematology consultation and BM harvest occurring only in the cell-treatment group),preoperative values will be established/confirmed and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria.However, individuals at high-risk, primarily neonates and those with preoperative advanced right heart failure will NOT be included such that the risk of this add-on procedure may not be acceptable. Following surgical Ebstein repair for the control group and following cell-based product delivery for the treatment group, all subjects will be followed for 24-months according to a pre-determined schedule that includes imaging studies along with a questionnaire, electrophysiology and laboratory studies at 1, 6, and 24 months post procedure. All subjects will be contacted for surveillance by phone at 3, 12, and 18 month post procedure.

Conditions

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Ebstein Anomaly

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment arm

Individuals with Ebstein anomaly and underlying myopathic right ventricle undergoing planned surgical intervention using an add-on procedure delivering autologous bone marrow-derived mononuclear cells into the right ventricle.

Group Type EXPERIMENTAL

Autologous Bone Marrow-derived Mononuclear Cells

Intervention Type BIOLOGICAL

Insertable cardiac monitor

Intervention Type DEVICE

Following surgical Ebstein repair a Medtronic Reveal LINQ Insertable Cardiac Monitor (ICM) will be placed subcutaneously in the anterior chest to continuously monitor the subject's heart rhythm and to record cardiac information through automatic detection of arrhythmias.

Control arm

Individuals with Ebstein anomaly and underlying myopathic right ventricle undergoing planned surgical intervention without cell delivery.

Group Type OTHER

Insertable cardiac monitor

Intervention Type DEVICE

Following surgical Ebstein repair a Medtronic Reveal LINQ Insertable Cardiac Monitor (ICM) will be placed subcutaneously in the anterior chest to continuously monitor the subject's heart rhythm and to record cardiac information through automatic detection of arrhythmias.

Interventions

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Autologous Bone Marrow-derived Mononuclear Cells

Intervention Type BIOLOGICAL

Insertable cardiac monitor

Following surgical Ebstein repair a Medtronic Reveal LINQ Insertable Cardiac Monitor (ICM) will be placed subcutaneously in the anterior chest to continuously monitor the subject's heart rhythm and to record cardiac information through automatic detection of arrhythmias.

Intervention Type DEVICE

Other Intervention Names

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MNC ICM ILR

Eligibility Criteria

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Inclusion Criteria

* Age 6 months to 30 years
* Individuals clinically planned for elective surgical Ebstein repair
* Individuals able to undergo bone marrow aspirate according to clinical consultation with Hematology (cell treatment group only)
* Individuals able to undergo preoperative MRI or CT examination
* Individual and/or parent willing and able to give informed consent and willing to commit to completion of follow-up

Exclusion Criteria

* Individuals requiring cavopulmonary shunt at the time of surgical Ebstein repair; planned preoperatively or required intraoperatively
* Individuals with, or reasonably expected to have, complications during surgical Ebstein repair or during post-operative recovery
* Individual who have not completed or will not be completing all pre-procedure work-up within 30 days of surgical Ebstein repair AND lack of pre-procedure work-up documented as a safety concern by a site investigator
* Individuals who have other clinical concerns as documented by a site investigator that could reasonably increase the risk of complications during or after surgical Ebstein repair
* Individuals whose cells have been determined, by the sponsor, to not be acceptable for release to the investigational site or individual whose cells have been compromised after cells released to investigational site (cell treatment group only)
* Individuals who require surgery on pulmonary, mitral, or aortic valve
* Individuals with pulmonary atresia or atrioventricular discordance with ventriculoarterial discordance
* Individuals with history of ventricular arrhythmia or new onset ventricular arrhythmia after enrollment that requires medical management
* Individuals who have undergone previous sternotomy
* Individuals with preoperative ventricular arrhythmia requiring medical management
* Individuals with severe chronic diseases, extensive extra-cardiac syndromes, or history of any cancer
* Individuals with current IV inotrope requirements
* Individuals with bleeding disorders or history of thrombosis
* Subjects not eligible for MRI or CT examination due to either a medical contraindication, including acute or chronic renal failure
* Individuals with a currently active infection being treated with oral antibiotics
* Individuals with the following conditions within 60 days prior to Ebstein repair surgery:

* Cardiogenic shock or extracorporeal circulation
* Documented infection requiring treatment with intravenous antibiotics
* Cardiac condition requiring emergency procedure
* Current or uncontrolled seizures or neurological injury that has resulted in a persistent deficit
* Multi-system organ failure including acute or chronic renal failure
* Female subjects 10 years and older with positive pregnancy test or lack of effective birth control method during the 30 days prior to surgical Ebstein repair
* Individuals who weigh more than 90 kg

Minimum Eligible Age

6 Months

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No