the Effect of Cerebrolysin on Physical and Mental Functions of Down Syndrome
Status:
Completed
Trial end date:
2019-08-30
Target enrollment:
Participant gender:
Summary
Down syndrome is a genetic disorder that causes delay in both physical growth and mental
development. It is the most frequently reported chromosomal abnormality and the most common
genetic syndrome.
Down syndrome is caused by trisomy of all or part of the genetic material of human chromosome
21. It is now estimated that 94% of individuals with Down syndrome have an extra chromosome
21 as a result of meiotic non-disjunction, or the abnormal segregation of chromosomes during
maternal gamete formation and of the remaining 5%, less than 1% is due to somatic mosaicism
and the rest is due to chromosome 21 translocations.
The estimated incidence of Down syndrome is between 1 / 1,000 to 1 / 1,100 live births
worldwide. In Egypt, the incidence of Down syndrome has been reported to be 1 / 1000 live
births.
Down syndrome is characterized by intellectual disability, short stature, distinctive facial
characters and a number of co-morbidities including cardiac and digestive anomalies, thyroid
problems, and childhood leukemia.
Down syndrome infants will likely experience delays in certain areas and aspects of
development. However, they will achieve all of the same milestones as other normal children,
just on their own timetable.
According to recent studies, the Down syndrome behavioral phenotype includes relative
strengths in some aspects of visuo-spatial processing and social functioning as well as
relative deficits in verbal processing. Language has been described as a "major area of
deficit" in Down syndrome individuals with particular difficulties manifested in expressive
language.
Due to this high incidence of Down syndrome in Egypt and the associated co-morbidities,
governmental care directed to this syndrome and other handicapping conditions has increased
tremendously in the past few years to the extent that Down syndrome phenotype has become a
phobia and many parents and/or physicians referred normal babies for karyotype due to either
suspicion of chromosomal anomalies or just for reassurance of their parents.
Although there has been enormous progress in the management of the physical aspects of Down
syndrome e.g. repair of heart defects, little advancement has been made to prevent
deterioration of cognitive function in these individuals. As a result, the dramatic increase
in life expectancy of children with Down syndrome in the past few decades has not been
paralleled with concurrent treatment for cognitive disabilities. Therefore, it has remained
the most common cause of cognitive dysfunction in children.
The pathogenesis of cognitive deficits and motor disabilities in Down syndrome individuals
can be attributed to diminished number and size of neuronal density, progressive neuronal
degeneration, impairment of neurogenesis, and reduction in dendrite formation as well as
spine density which results in disruption of synaptic function and plasticity. Therefore,
many of these individuals develop increasing problems with learning and memory in later life.
Cerebrolysin® is a neurotrophic peptidergic mixture isolated from pig brain. It is produced
by standardized enzymatic breakdown of lipid-free porcine brain proteins .
It acts similar to endogenous neurotrophic factors in the form of promoting neuronal
sprouting, stimulating neurogenesis, enhancing neuronal plasticity, and improving learning
and memory.
Several studies demonstrated that Cerebrolysin® can be used safely in the management of
children with any of the following medical conditions: minimal cerebral dysfunction,
resistant forms of nocturnal enuresis, neurosensory hypoacusis, attention deficit
hyperkinetic disorder, autism and Asperger syndrome.
The overall aim of the study is to assess the effect of Cerebrolysin® on neurocognitive
development of infants with Down syndrome.