NCT02549625

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2015

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2015

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

September 10, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 15, 2015

Completed
5.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2020

Completed
Last Updated

May 14, 2021

Status Verified

May 1, 2021

Enrollment Period

5.3 years

First QC Date

September 10, 2015

Last Update Submit

May 12, 2021

Conditions

Congenital Heart Disease

Keywords

Single right ventricular failure

Outcome Measures

Primary Outcomes (1)

  • Number of related serious adverse events

    6 months post cell delivery

Secondary Outcomes (12)

  • Number of subjects who died

    6 months post cell delivery

  • Number of subjects with sustained or symptomatic ventricular arrhythmias

    6 months post cell delivery

  • Number of subjects with acute decompensated heart failure

    6 months post cell delivery

  • Number of subjects with myocardial infarction

    6 months post cell delivery

  • Number of subjects with cardiac infection

    6 months post cell delivery

  • +7 more secondary outcomes

Study Arms (1)

Single-arm

EXPERIMENTAL

Intracoronary delivery of autologous bone marrow-derived mononuclear cells using catheterization procedure

Biological: Autologous Bone Marrow-derived Mononuclear Cells

Interventions

Autologous Bone Marrow-derived Mononuclear CellsBIOLOGICAL
Also known as: MNC
Single-arm

Eligibility Criteria

Age2 Years - 30 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Links

MeSH Terms

Conditions

Heart Defects, Congenital

Condition Hierarchy (Ancestors)

Cardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Timothy J Nelson, M.D., Ph.D.

    Mayo Clinic

    STUDY DIRECTOR

  • Muhammad Y Qureshi, MBBS

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Program Director

Study Record Dates

First Submitted

September 10, 2015

First Posted

September 15, 2015

Study Start

August 1, 2015

Primary Completion

November 1, 2020

Study Completion

November 1, 2020

Last Updated

May 14, 2021

Record last verified: 2021-05

Data Sharing

IPD Sharing
Will not share

Locations

US(1)