NCT01883076

Brief Summary

This is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of Hypoplastic Left Heart Syndrome (HLHS) children undergoing a scheduled Glenn surgical procedure. The investigators are doing this research study to find out if autologous stem cells from the individual's own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment for the management of HLHS.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
30

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started May 2013

Longer than P75 for phase_1

Geographic Reach
1 country

6 active sites

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

May 15, 2013

Completed
27 days until next milestone

First Submitted

Initial submission to the registry

June 11, 2013

Completed
10 days until next milestone

First Posted

Study publicly available on registry

June 21, 2013

Completed
7.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 28, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 28, 2021

Completed
Last Updated

April 29, 2022

Status Verified

April 1, 2022

Enrollment Period

8 years

First QC Date

June 11, 2013

Last Update Submit

April 28, 2022

Conditions

Hypoplastic Left Heart Syndrome

Keywords

Hypoplastic Left Heart SyndromeHLHSCongenital Heart DiseaseUmbilical cord bloodUCBCord bloodStem cellsRegenerative therapyStage II GlennGlenn Surgery

Outcome Measures

Primary Outcomes (4)

  • Incidence of all-cause mortality

    Within 2 years following cell therapy treatment

  • Incidence of new and worsening adverse cardiac events

    The adverse cardiac events would include sustained/symptomatic ventricular arrhythmias, heart failure, myocardial infarction, cardiac infections, and unexpected cardiovascular surgery.

    Within 2 years following cell therapy treatment

  • Percentage of subjects whose cells meet all cell release criteria

    Up to 2 years

  • Percentage of subjects enrolled who undergo cell therapy treatment

    Up to 2 years

Secondary Outcomes (9)

  • Change in right ventricular ejection fraction at one month according to cardiac imaging with echocardiography

    baseline, 1 month

  • Change in right ventricular ejection fraction at 3 months according to cardiac imaging with echocardiography

    baseline, 3 months

  • Change in right ventricular ejection fraction at 6 months according to cardiac imaging with echocardiography

    baseline, 6 months

  • Change in right ventricle tricuspid annular plane systolic excursion (TAPSE) at one month according to cardiac imaging with echocardiography

    baseline, 1 month

  • Change in right ventricle TAPSE at 3 months according to cardiac imaging with echocardiography

    baseline, 3 months

  • +4 more secondary outcomes

Study Arms (1)

autologous cell-based delivery

EXPERIMENTAL

autologous cell-based delivery a target dose of 3 million cells / kg of body weight will be delivered into the right heart muscle at the time of surgery. Cells are derived from autologous (self) umbilical cord blood.

Biological: autologous cell-based delivery

Interventions

autologous cell-based deliveryBIOLOGICAL

autologous cells (derived from "self")

Also known as: umbilical cord blood derived mononuclear cells
autologous cell-based delivery

Eligibility Criteria

AgeUp to 18 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Individuals with autologous cord blood product that met all cell release criteria (listed on the certificate of analysis from Mayo Clinic Human Cell Therapy Lab) as follows:
  • No aerobic or anaerobic bacterial growth after 14 days
  • Greater than 70% cell viability pre-freeze
  • Total Nucleated Cells (TNC) concentration of 30-42 x 106 cells/mL (pre-freeze)
  • Minimum of one (1) vial of cells
  • Mononuclear cell percentage of greater than 50%
  • Endotoxin result of less than 16 Endotoxin Units (EU)/mL.
  • Mother's serology test results are negative for HIV, Hepatitis B, and Hepatitis C.
  • Individuals with HLHS having undergone Stage I surgical palliation and undergoing planned Stage II palliative Glenn surgery.
  • Ages up to 18 months are eligible if written informed consent can be obtained from both parents (unless one parent is not reasonably available) and/or legal guardians.

You may not qualify if:

  • History of dimethyl sulfoxide (DMSO) reaction for either the child or mother.
  • Parent(s)/child unwilling to participate.
  • Child with severe chronic diseases, extensive extra-cardiac syndromic features, or history of cancer.
  • Child not completing all pre-procedure work-up within 10 days of the Stage II Glenn surgery as listed in section 6 of this protocol AND lack of pre-procedure work-up documented as a safety concern by a site investigator.
  • Child with the following complications of their congenital heart disease:
  • Any condition requiring urgent, or unplanned procedure within 15 days prior to Stage II surgical repair
  • Severe pulmonary hypertension (reported in the medical record as \>70% systemic pressure)
  • Other clinical concerns as documented by a site investigator that would predict (more likely to happen than not to happen) a risk of severe complications or very poor outcome during or after Stage II surgical repair.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's Hospital of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Oklahoma University Children's Hospital

Oklahoma City, Oklahoma, 73104, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Links

MeSH Terms

Conditions

Hypoplastic Left Heart SyndromeHeart 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

  • Harold M Burkhart, M.D.

    Oklahoma University Children's Hospital

    PRINCIPAL INVESTIGATOR

  • Joseph W Rossano, M.D.

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

  • David M Overman, M.D.

    Children's Hospital of Minnesota

    PRINCIPAL INVESTIGATOR

  • Ram Kumar Subramanyan, M.D., Ph.D.

    Children's Hospital Los Angeles

    PRINCIPAL INVESTIGATOR

  • James Jaggers, M.D.

    Children's Hospital Colorado

    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

June 11, 2013

First Posted

June 21, 2013

Study Start

May 15, 2013

Primary Completion

April 28, 2021

Study Completion

April 28, 2021

Last Updated

April 29, 2022

Record last verified: 2022-04

Data Sharing

IPD Sharing
Will not share

Locations

US(6)