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Hypoplastic Left Heart SyndromePhase 1NCT01883076

Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome

A Phase 1 study testing autologous cell-based delivery in people with Hypoplastic Left Heart Syndrome, run by Timothy J Nelson, MD, PhD at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages Up to 18 months
Conditions the study is for
Hypoplastic Left Heart Syndrome
What is being tested?
autologous cell-based delivery
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
6 sitesUnited States
Study dates
15 May 2013to 28 Apr 2021
Phase
Phase 1
Are there results?
None posted in the registry

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 18 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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%
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  • 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.

Who cannot join

  • Child who's UCB does not meet the specified cell release criteria in Inclusion Criterion #1.
  • 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 who's cells have been compromised after meeting cell release criteria (as defined in Inclusion Criterion #1).
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  • 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.

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • Children's Hospital of Minnesota

    Minneapolis, Minnesota, 55404

  • Oklahoma University Children's Hospital

    Oklahoma City, Oklahoma, 73104

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Mayo Clinic

    Rochester, Minnesota, 55905

Other trials in United States

About this study

Official title: Phase I Safety Study of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Stage II Palliation of Hypoplastic Left Heart Syndrome

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.

Read the detailed description

This study is a Phase I trial to determine the safety of autologous mononuclear cells (MNC) derived from umbilical cord blood for intramyocardial delivery into the right ventricle during a planned and non-emergent Stage II surgical palliation in subjects with HLHS. This is the first critical step towards applying autologous MNC therapy as an add-on regenerative intervention for congenital heart disease management. The choice of HLHS as the target disease for regenerative therapies in congenital heart disease management is multi-factorial and includes the following considerations: 1) Severity of of this incurable disease, 2) palliative nature and burden of long-term outcomes with a single right ventricular system, 3) three stages of planned surgical procedures that provide time points to adjunctively intervene, and 4) prenatal diagnosis enabling planned collection of UCB. An emerging goal for cardiac regeneration includes the application of cell-based technology to congenital heart disease, which is a favorable substrate due to the lack of fibrotic scaring, and the presence of a microenvironment that is expected to support ongoing cardiac proliferation and growth for functional remuscularization. This Phase I safety study will determine the feasibility of collection, processing, and delivery of autologous cells as used in adult cardiac regenerative protocols in the setting of HLHS surgical management.

Being tested
autologous cell-based delivery
Keywords
Hypoplastic Left Heart Syndrome, HLHS, Congenital Heart Disease, Umbilical cord blood, UCB, Cord blood, Stem cells, Regenerative therapy, Stage II Glenn, Glenn Surgery
Medical subject headings
Hypoplastic Left Heart Syndrome, Heart Defects, Congenital

What participants receive

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

  • autologous cell-based deliveryBiological

    Also called umbilical cord blood derived mononuclear cells

    autologous cells (derived from "self")

Study groups in NCT01883076
Study groupRoleWhat they receive
autologous cell-based deliveryExperimentalautologous 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.
Receives: Biological: autologous cell-based delivery

What is being measured

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

  1. Incidence of all-cause mortality

    Measured: Within 2 years following cell therapy treatment

  2. 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.

    Measured: Within 2 years following cell therapy treatment

  3. Percentage of subjects whose cells meet all cell release criteria

    Measured: Up to 2 years

  4. Percentage of subjects enrolled who undergo cell therapy treatment

    Measured: Up to 2 years

Secondary outcomes (9)

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

    Measured: baseline, 1 month

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

    Measured: baseline, 3 months

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

    Measured: baseline, 6 months

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

    Measured: baseline, 1 month

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

    Measured: baseline, 3 months

  6. Change in right ventricle TAPSE at 6 months according to cardiac imaging with echocardiography

    Measured: baseline, 6 months

  7. Change in right ventricle fractional area change at one month according to cardiac imaging with echocardiography

    Measured: baseline, 1 month

  8. Change in right ventricle fractional area change at 3 months according to cardiac imaging with echocardiography

    Measured: baseline, 3 months

  9. Change in right ventricle fractional area change at 6 months according to cardiac imaging with echocardiography

    Measured: baseline, 6 months

Timeline

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

  1. First posted21 Jun 2013 (estimated)
  2. Study start15 May 2013
  3. Primary completion28 Apr 2021
  4. Study completion28 Apr 2021

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01883076
Sponsor's protocol ID
12-008521
Status last verified
Apr 2022
First submitted
11 Jun 2013
Last updated
29 Apr 2022
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing autologous cell-based delivery in people with Hypoplastic Left Heart Syndrome, run by Timothy J Nelson, MD, PhD at 6 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 10 inclusion and 10 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: finished, as of the sponsor's last update on 29 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Incidence of all-cause mortality (time frame: Within 2 years following cell therapy treatment). 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 NCT01883076.

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 29 Apr 2022.

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