poster presentation

SMA Infant Biomarker Study – Super Baby Success!!
Stephen J. Kolb, M.D.,Ph.D., Christopher S. Coffey, Ph.D., Jon W. Yankey, Ph.D., W. David Arnold, M.D., Seward B. Rutkove, M.D., Amy Bartlett,
CCRC,. John T. Kissel, M.D., the NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators
The goal of the SMA Biomarker Study was to identify laboratory
measurements that will improve SMA clinical trials.
Enrollment began in November, 2012 and ended September, 2014. Enrollment of all
healthy control infants (27 infants younger than 6 months of age) was completed in less
than 1 year. Enrollment of infants with SMA (26 infants younger than 6 months of age)
also occurred ahead of schedule. Retention was excellent, and we have learned
essential clinical trial lessons from the Super Babies. Yay!!
Data collected in this study is important because…
ü  The data will accelerate the discovery and approval of SMA
therapies
ü  The data will improve the design of future SMA clinical trials
ü  The data will improve the design of future neonatal and
pediatric clinical studies
ü  The data will lead to specific questions about the biology of
SMA which will result in a better understanding of the disease
and better treatments for all people with SMA…. and is already
doing all of these things! J
Super Babies (and their Super Parents) volunteered to have their motor function tested,
to have electrical tests of the motor neurons and muscles performed and to have their
blood drawn for the first two years of life so that we can learn about how all of these
measurements change with time.
A specific Recruitment and Retention Plan was
developed prior to enrollment including our
informative YouTube™ video. Just type “SMA
Biomarker Video” into your search engine to
learn more.
The Super Babies Did It!
We are grateful to the parents and their infants for their
participation in this study. We also acknowledge the hard
work and dedication of the 15 NeuroNEXT site
coordinators, PIs and physical therapists who served as
Enrolling Sites for this multicenter study. Finally, we are
grateful to NINDS and the members of the Clinical
Coordinating Center (Mass General Hospital) and Data
Coordinating Center (University of Iowa) for their
continued support and enthusiasm for this project
We learned how motor function changes in infants
We learned how biomarkers relate to motor function
This study originates from The Ohio State University Wexner
Medical Center and Nationwide Children’s Hospital in
Columbus, OH
ü  We learned how relevant physiological and molecular biomarkers
change with time
ü  We learned how biomarkers CORRELATE with motor function
ü  We learned how biomarkers PREDICT future clinical outcomes
ü  We learned which tests are important and which are probably not
ü  We learned lessons about how best to design SMA clinical trials so
that current and future trials will have a higher chance of success
ü  We learned that Super Babies rock!
The SMA Infant Biomarker
Study is the premier study
of the NIH-Network for
Excellence in Neuroscience
Clinical Trials
This study is sponsored by the NINDS (U01-NS079163 to
SJK), CureSMA, the Muscular Dystrophy Association and
receives support from MDA, SMAF and WryBaby.com