Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT01856049 |
| Other study ID # |
11-007176 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
May 2012 |
| Est. completion date |
March 2025 |
Study information
| Verified date |
January 2024 |
| Source |
Mayo Clinic |
| Contact |
Lori A Riess |
| Phone |
507-538-7730 |
| Email |
riess.lori[@]mayo.edu |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
Cell-based cardiac regeneration has been the focus of acquired, adult heart disease for many
years. However, congenital heart disease with severe structural abnormalities may also be
reasonable targets for cell-based therapies. Interestingly, the pediatric heart is naturally
growing and may be the most amendable to regenerative strategies. Therefore, identifying
autologous cells (cells from the patient's own body) would be important to initiate these
studies.
This study aims to validate the use of umbilical cord blood as a source of autologous cells
for the purpose of cardiac repair of congenital heart disease. Cells will be isolated from
the cord blood to help us determine the feasibility of collection, processing, and storage of
these samples at the time of birth of infants with prenatal diagnosis of hypoplastic left
heart syndrome. This study may be useful for the development of pre-clinical and clinical
studies aimed at the long-term goal of repairing damaged heart muscle.
Description:
Congenital heart disease (CHD) is an abnormal formation that occurs during the development of
a baby's heart, heart valves and/or large vessels such as the aorta artery. CHD is the most
common cause of major congenital defects accounting for almost 30% of all defects. While the
statistics vary among studies, the best birth prevalence estimate is 8 per 1000 live births.
In the USA, CHD affects 1% of all births per year, with an estimated 40,000 babies born with
any type of heart defect every year.
The important improvements in CHD diagnosis and surgical treatment in the last decades has
led to an increased survival of newborns affected with heart defects. A large number of CHD
can be diagnosed during pregnancy, and the patients can present a broad range of symptoms.
Forms of CHD are usually classified based on their severity, from mild to severe. One of the
mildest forms of CHD is atrial septal defect, which can be undetectable until adulthood and
VSD. On the other hand, severe CHD that requires multiple palliative surgeries includes
single ventricle defects, such as hypoplastic left heart syndrome (HLHS), and tricuspid
atresia.
The survival of infants with CHD will depend on the severity of the defect and the time of
diagnosis and treatment received. The one-year survival of newborns with severe or critical
CHD (generally any type of surgery/procedures in their first year of life) is estimated to be
75%.
Stem cell therapy has emerged as a new paradigm of treatment in the field of CHD with
promising results. Cardiac regeneration has been the focus of acquired, adult heart disease
for many years. However, congenital heart disease with structural abnormalities may also be a
good target for other research studies. In fact, the pediatric heart is naturally growing and
may be amendable to regenerative strategies. Furthermore, the initial pre-clinical and
clinical studies have demonstrated that the delivery of stem cells into the heart of patients
with CHD is feasible and safe. Moreover, the cell therapy approach, along with the standard
surgical palliation, seems to offer benefits over surgical treatment alone. Even though the
number of cell therapy clinical trials for CHD has increased in the last decade, more
long-term follow-up studies are needed in this population setting in order to define the role
of stem cell therapy in the clinical practice. Therefore, confirming our ability to produce
autologous cells (cells from the patient's own body) from patients with severe CHD is an
important step towards the long-term goal of being able to discover innovative cell-based
protocols.
