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Hypoplastic Left Heart SyndromePhase 2NCT03779711

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

A Phase 2 study testing Autologous (self) mononuclear cells derived from umbilical cord blood and Stage II Surgical repair in people with Hypoplastic Left Heart Syndrome, run by Timothy J Nelson, MD, PhD at 8 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages Up to 12 months
Conditions the study is for
Hypoplastic Left Heart Syndrome
What is being tested?
Autologous (self) mononuclear cells derived from umbilical cord blood and Stage II Surgical repair
Could I receive a placebo?
No, everyone receives an active treatment
How many people
95 participants
Where
8 sitesUnited States
Study dates
6 Jun 2019to Feb 2026
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 12 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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)

Who cannot join

  • 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:
Show all 9Show fewer
  • 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.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites
  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Children's of Alabama

    Birmingham, Alabama, 35233

  • Cincinnati Children's Hospital and Medical Center

    Cincinnati, Ohio, 45529

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027

  • Children's Hospitals of Minnesota

    Minneapolis, Minnesota, 55404

  • Ocshner Medical Center

    New Orleans, Louisiana, 70121

  • Oklahoma University Medical Center

    Oklahoma City, Oklahoma, 73104

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official 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)

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.

Read the detailed description

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.

Being tested
Autologous (self) mononuclear cells derived from umbilical cord blood, Stage II Surgical repair
Keywords
Hypoplastic Left Heart Syndrome, HLHS, Congenital Heart Disease, Umbilical Cord Blood, UCB, Cord blood, Stem cells, Regenerative therapy, Stage II Glenn, Glenn Surgery, single ventricle
Medical subject headings
Hypoplastic Left Heart Syndrome, Heart Defects, Congenital, Univentricular Heart

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autologous (self) mononuclear cells derived from umbilical cord bloodBiological

    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 repairProcedure or surgery

    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.

Study groups in NCT03779711
Study groupRoleWhat they receive
TreatmentExperimentalAutologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 1-3 million cells per kilogram body weight . This is a one time treatment at the time of Stage II Glenn surgery .
Receives: Biological: Autologous (self) mononuclear cells derived from umbilical cord blood; Procedure: Stage II Surgical repair
ControlActive comparatorClinical data will be collected before, during and after the Stage II surgical standard of care procedure. Requires additional imaging studies at 3 months that are not standard of care in addition to some blood tests that would be beyond standard of care. Information will be collected to document the clinical outcomes and will be compared with the information from the group receiving the investigational treatment used in this study.
Receives: Procedure: Stage II Surgical repair

What is being measured

The primary outcome is the main question the study is designed to answer.

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

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

    Measured: baseline, 3 months post-Stage II surgery

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

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

    Measured: baseline, 3 months post-Stage II surgery

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

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

    Measured: baseline, 3 months post-Stage II surgery

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

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

    Measured: baseline, 12 months post-Stage II surgery

Secondary outcomes (12)
  1. Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC)

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

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

  2. Cumulative Days of Hospitalization

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

    Measured: 1-month post Stage II surgery

  3. Change in Weight

    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.

    Measured: baseline, 3-months post Stage II surgery

  4. Change in Heart Rate

    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.

    Measured: baseline, 3-months post Stage II surgery

  5. Change in Oxygen Saturation

    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.

    Measured: baseline, 3-months post Stage II surgery

  6. Change in Weight

    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.

    Measured: baseline, every 6 months post-Stage II surgery

  7. Change in Arrhythmia and Heart Failure Medication

    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.

    Measured: 3-months post-surgery

  8. Stage III Surgical Repair

    The number of research subjects eligible for Stage III surgical repair

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

  9. Total Time Until Listed for Cardiac Transplantation

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

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

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

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

    Measured: baseline, Stage III surgery pre-op

  11. Total Time Until Death

    The number of days between Stage II surgery and death occurrence

    Measured: approx 4 years

  12. Cumulative Days of Hospitalization

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

    Measured: 3-months post Stage II surgery

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Dec 2018
  2. Study start6 Jun 2019
  3. Primary completion22 May 2022
  4. Study completionFeb 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
95 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03779711
Sponsor's protocol ID
18-004753
Status last verified
Jun 2025
First submitted
4 Dec 2018
Last updated
8 Jul 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Gallego-Navarro C, Jaggers J, Burkhart HM, Carlo WF, Morales DL, Qureshi MY, Rossano JW, Hagen CE, Seisler DK, Peral SC, Nelson TJ. Autologous umbilical cord blood mononuclear cell therapy for hypoplastic left heart syndrome: a nonrandomized control trial of the efficacy and safety of intramyocardial injections. Stem Cell Res Ther. 2025 May 1;16(1):215. doi: 10.1186/s13287-025-04316-3.

    PubMed 40312733

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Autologous (self) mononuclear cells derived from umbilical cord blood and Stage II Surgical repair in people with Hypoplastic Left Heart Syndrome, run by Timothy J Nelson, MD, PhD at 8 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 8 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 8 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC) (time frame: baseline, 3 months post-Stage II surgery). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03779711, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2025.

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