- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01883076
Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Anticipated)
Phase
- Phase 1
Contacts and Locations
Study Locations
-
-
California
-
Los Angeles, California, United States, 90027
- Children's Hospital Los Angeles
-
-
Colorado
-
Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
-
-
Minnesota
-
Minneapolis, Minnesota, United States, 55404
- Children's Hospital of Minnesota
-
Rochester, Minnesota, United States, 55905
- Mayo Clinic
-
-
Oklahoma
-
Oklahoma City, Oklahoma, United States, 73104
- Oklahoma University Children's Hospital
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19104
- Children's Hospital of Philadelphia
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria
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%
- 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.
Exclusion Criteria
- 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).
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: autologous cell-based delivery
autologous 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.
|
autologous cells (derived from "self")
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of all-cause mortality
Time Frame: Within 2 years following cell therapy treatment
|
Within 2 years following cell therapy treatment
|
|
Incidence of new and worsening adverse cardiac events
Time Frame: Within 2 years following cell therapy treatment
|
The adverse cardiac events would include sustained/symptomatic ventricular arrhythmias, heart failure, myocardial infarction, cardiac infections, and unexpected cardiovascular surgery.
|
Within 2 years following cell therapy treatment
|
Percentage of subjects whose cells meet all cell release criteria
Time Frame: Up to 2 years
|
Up to 2 years
|
|
Percentage of subjects enrolled who undergo cell therapy treatment
Time Frame: Up to 2 years
|
Up to 2 years
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in right ventricular ejection fraction at one month according to cardiac imaging with echocardiography
Time Frame: baseline, 1 month
|
baseline, 1 month
|
Change in right ventricular ejection fraction at 3 months according to cardiac imaging with echocardiography
Time Frame: baseline, 3 months
|
baseline, 3 months
|
Change in right ventricular ejection fraction at 6 months according to cardiac imaging with echocardiography
Time Frame: baseline, 6 months
|
baseline, 6 months
|
Change in right ventricle tricuspid annular plane systolic excursion (TAPSE) at one month according to cardiac imaging with echocardiography
Time Frame: baseline, 1 month
|
baseline, 1 month
|
Change in right ventricle TAPSE at 3 months according to cardiac imaging with echocardiography
Time Frame: baseline, 3 months
|
baseline, 3 months
|
Change in right ventricle TAPSE at 6 months according to cardiac imaging with echocardiography
Time Frame: baseline, 6 months
|
baseline, 6 months
|
Change in right ventricle fractional area change at one month according to cardiac imaging with echocardiography
Time Frame: baseline, 1 month
|
baseline, 1 month
|
Change in right ventricle fractional area change at 3 months according to cardiac imaging with echocardiography
Time Frame: baseline, 3 months
|
baseline, 3 months
|
Change in right ventricle fractional area change at 6 months according to cardiac imaging with echocardiography
Time Frame: baseline, 6 months
|
baseline, 6 months
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Muhammad Y Qureshi, MBBS, Mayo Clinic
- Study Director: Timothy J Nelson, M.D., Ph.D., Mayo Clinic
- Principal Investigator: Harold M Burkhart, M.D., Oklahoma University Children's Hospital
- Principal Investigator: Joseph W Rossano, M.D., Children's Hospital of Philadelphia
- Principal Investigator: David M Overman, M.D., Children's Hospital of Minnesota
- Principal Investigator: Ram Kumar Subramanyan, M.D., Ph.D., Children's Hospital Los Angeles
- Principal Investigator: James Jaggers, M.D., Children's Hospital Colorado
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 12-008521
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hypoplastic Left Heart Syndrome
-
Mayo ClinicRecruitingHypoplastic Left Heart Syndrome (HLHS)United States
-
Children's Hospital Medical Center, CincinnatiRecruitingHypoplastic Left Heart Syndrome (HLHS)United States, United Kingdom, Canada
-
Emory UniversityCompleted
-
Athena ZuppaCompletedHypoplastic Left Heart | Tetrology of Fallot | Heart VentricleUnited States
-
HealthCore-NERISuspendedCongenital Heart Disease | Hypoplastic Left HeartUnited States, Canada
-
Athena ZuppaCompletedTetralogy of Fallot | Tricuspid Atresia | Hypoplastic Left Heart
-
Nationwide Children's HospitalNational Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health... and other collaboratorsRecruitingHeart Diseases | Cardiovascular Diseases | Heart Defects, Congenital | Cardiovascular Abnormalities | HLH - Hypoplastic Left Heart Syndrome | DORV | DILV - Double Inlet Left Ventricle | Mitral Atresia | Tricuspid Atresia | Unbalanced AV Canal | Single-ventricleUnited States
-
Baylor College of MedicineCompletedCongenital Heart Disease | Cardiac Disease | Hypoplastic Left Heart | Cyanotic Congenital Heart DiseaseUnited States
-
Okayama UniversityTranslational Research Center for Medical Innovation, Kobe, Hyogo, JapanCompletedHypoplastic Left Heart Syndrome | Single Right Ventricle | Single Left VentricleJapan
-
Kevin HillCompletedHypoplastic Left Heart Syndrome | Hypoplastic Right-sided Heart ComplexUnited States
Clinical Trials on autologous cell-based delivery
-
Ministry of Health, BrazilHospital de Clinicas de Porto Alegre; Instituto de Cardiologia do Rio Grande... and other collaboratorsTerminated
-
James Baumgartner, MDCBR Systems, Inc.CompletedSensorineural Hearing LossUnited States
-
James Baumgartner, MDSuspendedSensorineural Hearing LossUnited States
-
Mayo ClinicUniversity of Alabama at Birmingham; University of Mississippi Medical CenterCompletedChronic Kidney Disease | Renal Artery Stenosis | Ischemic Nephropathy | Renovascular DiseaseUnited States
-
Florida State UniversityCompletedHIV InfectionsUnited States
-
Zhi-Hong Liu, M.D.Nanjing Medical University; Soochow UniversityCompleted
-
Maastricht UniversityRecruitingMitochondrial MyopathiesNetherlands
-
University of WashingtonNational Institute of Mental Health (NIMH)Completed
-
Northwestern UniversityTerminated
-
Jan WalewskiCompletedLymphoma | Multiple Myeloma and Plasma Cell NeoplasmPoland