NCT01445041

Brief Summary

Further study details as provided by Duke University: Purpose: To evaluate the feasibility and safety of collecting and infusing autologous umbilical cord blood (UCB) in newborn infants with hypoplastic left heart syndrome (HLHS). Study Rationale and Hypotheses: The major goal of this study is to determine whether infusion of autologous UCB cells in neonates with hypoplastic left heart syndrome is feasible and safe. The rationale for the study and for the potential benefit of UCB is based upon the following hypotheses:

  1. 1.Infants with HLHS have significant neural injury evidenced from both prenatal and early antenatal brain MRI findings and infusion of UCB cells may lessen neural injury. Although the exact mechanism is unknown, UCB cell infusion may ameliorate neural injury via paracrine and anti-inflammatory effects that enhance post injury repair and may promote endogenous functional compensation of other cortical areas resulting in significant clinical improvements.
  2. 2.UCB cells may also enhance cardiac function, minimize scar formation, and reverse detrimental remodeling after cardiac injury.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
7

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Sep 2011

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2011

Completed
14 days until next milestone

First Submitted

Initial submission to the registry

September 15, 2011

Completed
18 days until next milestone

First Posted

Study publicly available on registry

October 3, 2011

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 15, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 15, 2016

Completed
Last Updated

May 16, 2024

Status Verified

May 1, 2024

Enrollment Period

4.6 years

First QC Date

September 15, 2011

Last Update Submit

May 14, 2024

Conditions

Hypoplastic Left Heart Syndrome

Keywords

Hypoplastic Left Heart SyndromeAutologous Umbilical Cord BloodNewborn Infants

Outcome Measures

Primary Outcomes (1)

  • Adverse event rates occurring in the pilot study population. The investigators will compare infusion outcomes of infants infused with frozen cells and infants infused with non-frozen cells.

    During Infusions (First 2 months of life)

Secondary Outcomes (1)

  • Feasibility and preliminary efficacy

    1 year

Study Arms (2)

Single infusion of UCB

EXPERIMENTAL

(autologous red blood cell and volume reduced cord blood cells)

Biological: Autologous Umbilical Cord Blood

Three infusions of UCB

EXPERIMENTAL

(autologous red blood cell and volume reduced cord blood cells)

Biological: Autologous Umbilical Cord Blood

Interventions

Autologous Umbilical Cord BloodBIOLOGICAL

Infants who meet study enrollment criteria for hypoplastic left heart syndrome in the neonatal period will receive 1 infusion of their own volume reduced cord blood cells. The dose for each infusion is 5x10e7 cells/kg.

Single infusion of UCB

Eligibility Criteria

AgeUp to 2 Days
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Infants \> 35 weeks gestational age.
  • Diagnosis: Hypoplastic Left Heart Syndrome.
  • Autologous umbilical cord blood available with a minimum total nucleated cell dose of 1 x 10e7 cells/kg.
  • Parental Consent.

You may not qualify if:

  • Chromosomal anomalies identified before the time of infusion.
  • Chromosomal anomalies or congenital anomalies that would prohibit clinicians from initiating surgical repair of the congenital heart defect.
  • Infant is determined by clinical staff to be non-viable and will not receive aggressive care. (No member on the study team will be involved in determining the viability of the neonate.)
  • Autologous umbilical cord blood unit has any of the following:
  • Total nuclear cell count \< 1 x 10e7.
  • Positive maternal infectious serology (except CMV).
  • Evidence of infectious contamination of the cord blood unit.
  • Evidence of genetic disease.
  • Unable to obtain parental consent.
  • Mother \< 18 years old.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University

Durham, North Carolina, 27710, United States

Location

Related Publications (6)

  • Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. doi: 10.1016/j.bbmt.2005.09.016.

    PMID: 16443516BACKGROUND

  • Kurtzberg J, Lyerly AD, Sugarman J. Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood. J Clin Invest. 2005 Oct;115(10):2592-7. doi: 10.1172/JCI26690.

    PMID: 16200191BACKGROUND

  • Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.

    PMID: 15901860BACKGROUND

  • Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, Allison-Thacker J, Wood S, Wenger DA, Rubinstein P, Hopwood JJ, Krivit W, Kurtzberg J. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004 May 6;350(19):1960-9. doi: 10.1056/NEJMoa032613.

    PMID: 15128896BACKGROUND

  • McGraw P, Liang L, Escolar M, Mukundan S, Kurtzberg J, Provenzale JM. Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience. Radiology. 2005 Jul;236(1):221-30. doi: 10.1148/radiol.2353040716.

    PMID: 15987975BACKGROUND

  • Mahle WT, Tavani F, Zimmerman RA, Nicolson SC, Galli KK, Gaynor JW, Clancy RR, Montenegro LM, Spray TL, Chiavacci RM, Wernovsky G, Kurth CD. An MRI study of neurological injury before and after congenital heart surgery. Circulation. 2002 Sep 24;106(12 Suppl 1):I109-14.

    PMID: 12354718BACKGROUND

Related Links

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome

Condition Hierarchy (Ancestors)

Heart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Charles M Cotten, MD MHS

    Duke University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Associate Professor of Pediatrics

Study Record Dates

First Submitted

September 15, 2011

First Posted

October 3, 2011

Study Start

September 1, 2011

Primary Completion

April 15, 2016

Study Completion

April 15, 2016

Last Updated

May 16, 2024

Record last verified: 2024-05

Locations

US(1)