March 19th -
Clinical trials involving children are very rare, and even more so when it comes to low-prevalence diseases.
Dr. Rafael de la Torre, director of the Neurosciences research programme at the Hospital del Mar Medical Research Institute (IMIM), is leading a clinical trial involving a paediatric population, the aim of which is to evaluate the safety and effectiveness of a dietary preparation containing epigallocatechin gallate (EGCG) - a natural compound found in green tea- on improved cognitive development in children, aged 6 to 12, with Down's syndrome, and which will also evaluate its effect on children with Fragile X syndrome.
The paediatric clinical trials will take place simultaneously in 5 health centres: Hospital Niño Jesús (Madrid), Instituto Hispalense de Pediatria (Seville), Hospital Universitario Marqués de Valdecilla (Santander), Hospital del Mar ( Barcelona), and Institut Jérome Lejeune (Paris).
It is a randomised clinical trial that will involve about 70 children with Down's syndrome, and will also include children affected by Fragile X syndrome. "We are including children with Down's syndrome and Fragile X because, because although the genetic origin of the two syndromes is different, the mechanism through which the epigallocatechin gallate could provide therapeutic effects is the same, and for this reason we think that Fragile X sufferers could also benefit", adds Dr. de la Torre.
The recruitment phase for the study began in February 2018, and is expected to last 10 months. It is being funded by the Mutua Madrileña and Jérome Lejeune foundations and will be supported by Dr. Mara Dierssen, coordinator of the Cell Systems and Neurobiology group at the Centre for Genomic Regulation (CRG) and a specialist in the neurobiology of Down's syndrome. It is thanks to her pre-clinical studies that this clinical trial can go ahead.
The recruitment phase for the study began in February 2018, and is expected to last 10 months. It is being funded by the Mutua Madrileña and Jérome Lejeune foundations and will be supported by Dr. Mara Dierssen, coordinator of the Cell Systems and Neurobiology group at the Centre for Genomic Regulation (CRG) and a specialist in the neurobiology of Down's syndrome. It is thanks to her pre-clinical studies that this clinical trial can go ahead.
In 2016, the Hospital del Mar Medical Research Institute (IMIM) and Centre for Genomic Regulation (CRG) presented the results of a phase 2 clinical trial on EGCG, involving young adults aged between 16 and 34 who suffered Down's syndrome. The results demonstrated that the participants who had received treatment with EGCG and cognitive stimulation had improved their visual recognition memory and attention, as well as self-control and adaptive behaviour, and autonomy in daily activities, and that these changes could be correlated with biological modifications in brain connectivity. The study was published in the journal Lancet Neurology.
"It was the first time that any treatment demonstrated efficacy in improving cognitive functions in people with Down's syndrome, even though brain plasticity in the adult population is limited because the brain has already developed fully. These good results in young adults and the safety of the treatment have enabled this innovative clinical trial to be started in the paediatric population, which, thanks to the greater plasticity children's brains, we hope will give even better results and that these benefits will last a lot longer", explains Dr. Rafael de la Torre.
According to the World Health Organisation (WHO) Down's Syndromeaffects approximately 1 in 1,000 people across the globe and is the most common genetic cause of intellectual disability. It is caused by a trisomy of chromosome 21, in other words, people with this syndrome have three copies of chromosome 21, not two. Epigallocatechin gallate modulates the activity of one of the genes located on chromosome 21, DYRK1A, which is related to brain plasticity and certain cognitive abilities.
Fragile X syndrome is an inherited genetic disorder linked to the X chromosome, and can cause problems ranging from learning difficulties to decreased intellectual ability. It affects 1 in 4,000 boys and 1 in 6,000 girls.
"For 15 years, Fundación Mutua Madrileña has been firmly committed to clinical research carried out in Spain and each year supports the implementation of clinical studies aimed at improving treatments for diseases where needs are not being met. Studies like this, which aim to improve the cognitive abilities of children with Down's syndrome and Fragile X, are our raison d'être", says Lorenzo Cooklin, CEO of Fundación Mutua Madrileña.
"Jérôme Lejeune Institute is a French medical, research and training center for the scientific study of Down syndrome and others intellectual disabilities from genetic origin. In 2018, the Jérôme Lejeune Institute is involved in a dozen research programs, including the PERSEUS clinical research program as an investigation center. With more than 9 000 patients, of whom the majority are followed since birth, the Institute's consultation offers a unique and exceptional collection of experiences and clinical information for the understanding and treatment of Down syndrome, whose studies, such as PERSEUS, are the first blessings", says Sophie Durand, research director at Jérôme Lejeune Institute.
To facilitate the study, Laboratoires Grand Fontaine have developed a preparation that takes into account some of the more common metabolic problems seen in people with disabilities, such as coeliac disease and constipation, and they have worked on a format specially designed for the paediatric population. Laboratoires Grand Fontaine have donated the medication for this clinical study.
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