NCT01856049

Brief Summary

Cell-based cardiac regeneration has been the focus of acquired, adult heart disease for many years. However, congenital heart disease with severe structural abnormalities may also be reasonable targets for cell-based therapies. Interestingly, the pediatric heart is naturally growing and may be the most amendable to regenerative strategies. Therefore, identifying autologous cells (cells from the patient's own body) would be important to initiate these studies. This study aims to validate the use of umbilical cord blood as a source of autologous cells for the purpose of cardiac repair of congenital heart disease. Cells will be isolated from the cord blood to help us determine the feasibility of collection, processing, and storage of these samples at the time of birth of infants with prenatal diagnosis of hypoplastic left heart syndrome. This study may be useful for the development of pre-clinical and clinical studies aimed at the long-term goal of repairing damaged heart muscle.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
464

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started May 2012

Longer than P75 for not_applicable

Geographic Reach
1 country

2 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 1, 2012

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

May 14, 2013

Completed
3 days until next milestone

First Posted

Study publicly available on registry

May 17, 2013

Completed
11.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 12, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 12, 2024

Completed
Last Updated

January 10, 2025

Status Verified

January 1, 2025

Enrollment Period

12.5 years

First QC Date

May 14, 2013

Last Update Submit

January 8, 2025

Conditions

Hypoplastic Left Heart Syndrome (HLHS)

Keywords

HLHSHypoplastic Left Heart SyndromeUCB (umbilical cord blood)Umbilical cord bloodCord bloodCardiac regeneration

Outcome Measures

Primary Outcomes (2)

  • Percent of samples contaminated

    14 days after collection

  • Percent of cells that are viable following post thaw analysis

    5 years

Study Arms (1)

Umbilical Cord Blood Collection

OTHER

Umbilical Cord Blood is drawn from the umbilical cord of newborn babies diagnosed with Hypoplastic Left Heart Syndrome, before placental detachment. Cord blood is packaged in a Credo Cube, and sent at a temperate state to the manufacturer immediately after draw. At least 65 mL of cord blood is needed to produce a stem cell product during manufacturing. Once processed, the patient's autologous cord blood stem cells will be frozen for their potential future use in a clinical trial.

Other: Collection of umbilical cord blood

Interventions

Collection of umbilical cord bloodOTHER

Cord blood will be processed in the temperate state it was collected to produce a pure, stem cell product identifiable to patients with Hypoplastic Left Heart Syndrome, and will be stored at a frozen state for their potential, future use in a clinical trial.

Umbilical Cord Blood Collection

Eligibility Criteria

Sexfemale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Any pregnant woman, regardless of age, with a prenatal diagnosis of severe CHD/hypoplastic left heart syndrome.
  • One or both parents willing to consent to the storage of umbilical cord blood for the specific purpose of regenerative research
  • Delivering party and/or expectant family is willing to sign Release of Information to request fetal echo text report diagnosing severe CHD/hypoplastic left heart syndrome
  • Parent(s) willing to be contacted 60 days after collection for follow-up screening questions regarding the health status of the baby affected with severe CHD/hypoplastic left heart syndrome.

You may not qualify if:

  • Individuals unwilling to participate

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Links

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome

Condition Hierarchy (Ancestors)

Heart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Susana Cantero Peral, M.D., Ph.D.

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
PI

Study Record Dates

First Submitted

May 14, 2013

First Posted

May 17, 2013

Study Start

May 1, 2012

Primary Completion

November 12, 2024

Study Completion

November 12, 2024

Last Updated

January 10, 2025

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will not share

Locations

US(2)