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NCT Number: NCT03779711

Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Repair of Hypoplastic Left Heart Syndrome

Researchers want to better understand what happens to the heart when the stem cells are injected directly into the muscle of the right side of the heart during the Stage II palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS) or HLHS variant. Researchers want to see if there are changes in the heart's structure/function following this stem cell-based therapy and compared to children that have not had cell-based therapy.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Hypoplastic Left Heart Syndrome Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Heart Defects, Congenital Heart Diseases Univentricular Heart

Age range

Up to 12 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Children's of Alabama, Birmingham, Alabama, United States

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About this study

This is a Phase IIb trial to determine if the delivery of the autologous UCB-MNC product into the myocardium of the right ventricle of the heart at the time of Stage II surgical repair will provide an improvement in cardiac function, reaching growth and developmental milestones, and quality of life, while also providing a reduction in the cumulative days of hospitalization following Stage II surgical repair. Long-term improvement in cardiac function, reaching growth and developmental milestones, reaching Stage III surgical repair pre-op work-up, prolonging time to cardiac transplantation or death, and improving quality of life will also be determined.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of HLHS or HLHS variant with single right ventricular dependent CHD having undergone Stage I surgical repair and undergoing Stage II surgical repair.
  • Less than 13 months of age at time of Stage II surgical repair
  • Previous participation in the UCB collection protocol with autologous UCB-MNC product that is acceptable for use (treatment arm only)

Exclusion criteria

  • History of DMSO reaction (treatment arm only).
  • Parent(s) and/or legal guardian(s) unwilling to have their child participate or unwilling to follow the study procedures.
  • Severe chronic diseases at the discretion of the treating physician.
  • Extensive extra-cardiac syndromic features.
  • Known history of cancer.
  • Any of the following complications of his/her congenital heart disease:
  • Any condition requiring urgent, or unplanned interventional procedure within 15 days prior to Stage II surgical repair, unless complete and full cardiac recovery is documented by site investigator
  • 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, not directly related to the stem cell product or its injection procedure, during or after Stage II surgical repair.

Treatment and study plan

Autologous (self) mononuclear cells derived from umbilical cord blood

Biological

The investigational product will be delivered into the right myocardium via sub-epicardial injections of 0.1 mL per kg body weight to achieve the target dose of 1-3 million TNC per kg body weight.at the time of Stage II surgical repair.

Stage II Surgical repair

Procedure

This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.

Primary outcomes

  1. Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)

    Time frame: baseline, 3 months post-Stage II surgery

    Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.

  2. Change in Right Ventricular Cardiac Function Measured by Circumferential Strain.

    Time frame: baseline, 3 months post-Stage II surgery

    Right ventricular cardiac function as measured by echocardiogram that includes studies for circumferential strain. Measured by percent.

  3. Change in Right Ventricular Cardiac Function Measured by Longitudinal Strain.

    Time frame: baseline, 3 months post-Stage II surgery

    Right ventricular cardiac function as measured by echocardiogram that includes studies for longitudinal strain. Measured by percent.

  4. Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)

    Time frame: baseline, 12 months post-Stage II surgery

    Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.

Secondary outcomes

  1. Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)

    Time frame: baseline pre-op, hospital discharge from Stage II surgery (an average of 1-6 weeks)

    Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.

  2. Cumulative Days of Hospitalization

    Time frame: 1-month post Stage II surgery

    Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.

  3. Change in Weight

    Time frame: baseline, 3-months post Stage II surgery

    Change in weight between baseline and 3-months post Stage II surgery. Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.

  4. Change in Heart Rate

    Time frame: baseline, 3-months post Stage II surgery

    Change in heart rate between baseline and 3-months post Stage II surgery. Heart rate as measured at home by subject's caregiver as standard of care per subject's provider. Caregivers will be instructed by the site staff on the best way to perform.

  5. Change in Oxygen Saturation

    Time frame: baseline, 3-months post Stage II surgery

    Change in oxygen saturation between baseline and 3-months post Stage II surgery. Oxygen saturation as measured at home by subject's caregiver using the pulse oximetry device provided as standard of care by subject's provider. Caregivers will be instructed by the site to use the same pulse oximetry device for every home measurement of oxygen saturation.

  6. Change in Weight

    Time frame: baseline, every 6 months post-Stage II surgery

    Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.

  7. Change in Arrhythmia and Heart Failure Medication

    Time frame: 3-months post-surgery

    Changes in arrhythmia and heart failure medications will be recorded by caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.

  8. Stage III Surgical Repair

    Time frame: Stage III surgery pre-op, approx. 4 years

    The number of research subjects eligible for Stage III surgical repair

  9. Total Time Until Listed for Cardiac Transplantation

    Time frame: up to Stage III surgery pre-op, approx. 4 years

    The number of days between Stage II surgery and listed on cardiac transplantation list.

  10. Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)

    Time frame: baseline, Stage III surgery pre-op

    Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.

  11. Total Time Until Death

    Time frame: approx 4 years

    The number of days between Stage II surgery and death occurrence

  12. Cumulative Days of Hospitalization

    Time frame: 3-months post Stage II surgery

    Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.

Sponsors and collaborators

Lead sponsor

Timothy J Nelson, MD, PhD

Other

Collaborators

  • Children's Hospital Colorado
  • Children's Hospital Los Angeles
  • Children's Hospital Medical Center, Cincinnati
  • Children's Hospital of Philadelphia
  • Children's Hospitals and Clinics of Minnesota
  • Children's of Alabama
  • Ochsner Health System
  • University of Oklahoma

Registry information

Official study title

Phase IIb Study of Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Stage II Surgical Repair of Right Ventricular Dependent Variants of Hypoplastic Left Heart Syndrome (AutoCell-S2)

Important dates

Study start
2019
Primary completion
2022
Study completion
2026
First posted
Dec 19, 2018
Registry last updated
Jul 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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