Muscular Atrophy, Spinal Clinical Trial
Official title:
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and to detect the clinical efficacy of HU treatment in children with Types II and III SMA.
SMA is a neuromuscular disorder characterized by degeneration of spinal cord motor neurons
and muscular atrophy. SMA is classified into three clinical subtypes according to the
severity and age of onset (Types I, II and III). Type II (intermediate) SMA has its onset in
early childhood (prior to 18 months) and is characterized by the failure to stand or walk
unassisted. Individuals with Type III SMA (mild SMA or Kugelberg-Welander disease) typically
develop symptoms after 18 months of age and display a wide range of clinical heterogeneity.
The clinical spectrum ranges from rapid progressive weakness resulting in wheelchair
dependence in late childhood to patients being able to walk in adult years and living
productive and independent lifestyles for the majority of their lives.
In our laboratory, our preliminary results indicate that HU treatment significantly increases
both SMN mRNA expression and intact SMN protein levels in vitro. These data confirm previous
observations that in vitro treatments of SMA lymphocytes with hydroxyurea resulted in
augmentation of the SMN2 gene expression in a dose and time related manner. Based on these
exciting pre-clinical data, coupled with the well-documented side-effect profile of HU in
children, we are conducting a pilot clinical trial using HU in children with Types II and III
SMA. This clinical trial study is intended to establish the safety profile in children with
Types II and III SMA; to identify reliable outcome measures; and to detect the possible
clinical efficacy of HU treatment in children with Types II and III SMA.
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