Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells



Status:Active, not recruiting
Conditions:Peripheral Vascular Disease, Cardiology, Cardiology
Therapuetic Areas:Cardiology / Vascular Diseases
Healthy:No
Age Range:2 - 30
Updated:12/9/2018
Start Date:August 2015
End Date:November 2020

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Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells for Systemic, Single Right Ventricular Failure Due to Congenital Heart Disease

The objective of this study is to determine the safety and feasibility of autologous
mononuclear cells (MNC) collected from bone marrow (BM) and intracoronary delivery for
individuals with declining performance of their single right ventricle systemic pumps. This
procedure has the potential to foster a new strategy for congenital heart patients. This is
an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to
document 1) related serious adverse events and 2) monitor changes in cardiac structure and
function.

This study is an open label Phase I trial to determine the safety and feasibility of bone
marrow-derived mononuclear cells delivered into the coronary circulation of subjects with
Fontan circulation and right ventricular dysfunction. Subjects will be screened at outpatient
clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered
participation in this trial. Once informed consent has been obtained; preoperative values
will be established and a selection committee will review subjects within three days prior to
planned procedure to confirm inclusion and exclusion criteria. This will require a non-study
cardiologist to review the clinical case and confirm the baseline cardiac function with
evidence of disease progression with right ventricle dysfunction. However, individuals with
decreased cardiac function requiring inotropic support and immediate listing for cardiac
transplantation will NOT be included such that the risk of this research procedure may not be
acceptable. Following cell-based product delivery, subjects will be followed for 6-months
according to a pre-determined schedule that includes imaging studies along with
questionnaires, electrophysiology and laboratory studies. Subjects will be contacted for an
additional 18-months for surveillance by phone.

Inclusion Criteria:

- Individuals with Fontan circulation with right ventricular dysfunction

- Ejection fraction equal to or less than 40% despite optimal outpatient medical
management for at least 3 months

- Able to undergo bone marrow aspirate according to clinical consultation with
hematology (INR must be maintained at or below 1.5)

- Able to undergo an MRI or CT examination

Exclusion Criteria:

- Individuals or parents of minors unwilling to consent to participation

- Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer

- Females 10 years and older with positive pregnancy test or lack of effective birth
control method

- Individuals currently requiring IV inotropes

- Individuals with bleeding disorders or history of thrombosis

- Individuals not eligible for MRI and have elevated serum creatinine level (eGFR less
that 45 mL/min) and therefore cannot have a CT examination

- Individuals not eligible for MRI and have a prior significant reaction to intravenous
contrast required for CT examination

- Individuals with the following conditions within 60 days prior to procedure:

- Cardiogenic shock or extracorporeal circulation;

- New arrhythmia that required medication for control;

- Documented infection requiring treatment with antibiotics, and/or current
infection being treated with antibiotics;

- Cardiac condition requiring emergency procedure;

- Cardiovascular surgery;

- Seizures or history of significant neurological injury;

- Multi-system organ failure including acute or chronic renal failure
We found this trial at
1
site
Rochester, Minnesota 55905
Principal Investigator: Muhammad Y Qureshi, MBBS
Phone: 507-538-7730
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mi
from
Rochester, MN
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