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

• Register: ClinicalTrials.gov
• Last refreshed on: 9 May 2022
• Main ID: NCT01883076
• Date of registration: 11/06/2013
• Prospective Registration: No
• Primary sponsor: Timothy J Nelson, MD, PhD
• Public title: Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome
• Scientific title: Phase I Safety Study of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Stage II Palliation of Hypoplastic Left Heart Syndrome
• Date of first enrolment: May 15, 2013
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01883076
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Muhammad Y Qureshi, MBBS
• Affiliation: Mayo Clinic

• Name: Harold M Burkhart, M.D.
• Affiliation: Oklahoma University Children's Hospital

• Name: Joseph W Rossano, M.D.
• Affiliation: Children's Hospital of Philadelphia

• Name: Timothy J Nelson, M.D., Ph.D.
• Affiliation: Mayo Clinic

• Name: James Jaggers, M.D.
• Affiliation: Children's Hospital Colorado

• Name: Ram Kumar Subramanyan, M.D., Ph.D.
• Affiliation: Children's Hospital Los Angeles

• Name: David M Overman, M.D.
• Affiliation: Children's Hospital of Minnesota

Key inclusion & exclusion criteria

Inclusion Criteria

1. 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:

1. No aerobic or anaerobic bacterial growth after 14 days

2. Greater than 70% cell viability pre-freeze

3. Total Nucleated Cells (TNC) concentration of 30-42 x 106 cells/mL (pre-freeze)

4. Minimum of one (1) vial of cells

5. Mononuclear cell percentage of greater than 50%

6. Endotoxin result of less than 16 Endotoxin Units (EU)/mL.

2. Mother's serology test results are negative for HIV, Hepatitis B, and Hepatitis C.

3. Individuals with HLHS having undergone Stage I surgical palliation and undergoing
planned Stage II palliative Glenn surgery.

4. 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.

Exclusion Criteria

1. Child who's UCB does not meet the specified cell release criteria in Inclusion
Criterion #1.

2. History of dimethyl sulfoxide (DMSO) reaction for either the child or mother.

3. Parent(s)/child unwilling to participate.

4. Child with severe chronic diseases, extensive extra-cardiac syndromic features, or
history of cancer.

5. 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.

6. Child who's cells have been compromised after meeting cell release criteria (as
defined in Inclusion Criterion #1).

7. Child with the following complications of their congenital heart disease:

1. Any condition requiring urgent, or unplanned procedure within 15 days prior to
Stage II surgical repair

2. Severe pulmonary hypertension (reported in the medical record as >70% systemic
pressure)

3. 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.



Age minimum: N/A
Age maximum: 18 Months
Gender: All

Health Condition(s) or Problem(s) studied

Hypoplastic Left Heart Syndrome

Intervention(s)

Biological: autologous cell-based delivery

Primary Outcome(s)

Percentage of subjects whose cells meet all cell release criteria [Time Frame: Up to 2 years]

Incidence of all-cause mortality [Time Frame: Within 2 years following cell therapy treatment]

Incidence of new and worsening adverse cardiac events [Time Frame: Within 2 years following cell therapy treatment]

Percentage of subjects enrolled who undergo cell therapy treatment [Time Frame: Up to 2 years]

Secondary Outcome(s)

Change in right ventricle TAPSE at 6 months according to cardiac imaging with echocardiography [Time Frame: baseline, 6 months]

Change in right ventricle TAPSE at 3 months according to cardiac imaging with echocardiography [Time Frame: baseline, 3 months]

Change in right ventricular ejection fraction at 6 months according to cardiac imaging with echocardiography [Time Frame: baseline, 6 months]

Change in right ventricle fractional area change at 3 months according to cardiac imaging with echocardiography [Time Frame: baseline, 3 months]

Change in right ventricle fractional area change at 6 months according to cardiac imaging with echocardiography [Time Frame: baseline, 6 months]

Change in right ventricle fractional area change at one month according to cardiac imaging with echocardiography [Time Frame: baseline, 1 month]

Change in right ventricle tricuspid annular plane systolic excursion (TAPSE) at one month according to cardiac imaging with echocardiography [Time Frame: baseline, 1 month]

Change in right ventricular ejection fraction at 3 months according to cardiac imaging with echocardiography [Time Frame: baseline, 3 months]

Change in right ventricular ejection fraction at one month according to cardiac imaging with echocardiography [Time Frame: baseline, 1 month]

Secondary ID(s)

12-008521

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

University of Oklahoma

Children's Hospital Colorado

Mayo Clinic

Children's Hospital Los Angeles

Children's Hospital of Philadelphia

Children's Hospitals and Clinics of Minnesota