CompletedPhase 1

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

United States10 participantsStarted 2016-09

Plain-language summary

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.

Who can participate

Age range

6 Months – 30 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 discor…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of adverse events from time of index procedure

Timeframe: 24 months post index procedure

Severity of adverse events from time of index procedure

Timeframe: 24 months post index procedure

Number of subjects who died

Timeframe: 24 months post index procedure

Number of subjects with sustained symptomatic cardiac arrhythmias

Timeframe: 24 months post index procedure

Number of subjects with myocardial infarction

Timeframe: 24 months post index procedure

Number of subjects with unexpected, invasive cardiovascular procedures

Timeframe: 24 months post index procedure

Number of serious adverse events

Timeframe: 24 months post index procedure

Trial details

NCT IDNCT02914171

SponsorTimothy J. Nelson

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-03-18

View on ClinicalTrials.gov More Ebstein Anomaly trials

Plain-language summary

Who can participate

Age range

6 Months – 30 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Number of adverse events from time of index procedure

Timeframe: 24 months post index procedure

Severity of adverse events from time of index procedure

Timeframe: 24 months post index procedure

Number of subjects who died

Timeframe: 24 months post index procedure

Number of subjects with sustained symptomatic cardiac arrhythmias

Timeframe: 24 months post index procedure

Number of subjects with myocardial infarction

Timeframe: 24 months post index procedure

Number of subjects with unexpected, invasive cardiovascular procedures

Timeframe: 24 months post index procedure

Number of serious adverse events

Timeframe: 24 months post index procedure