![]() Because there is a suggestion of possible benefit in treated subjects, we recommend further study of riluzole in pediatric patients with SMA. This was a limited study with insufficient power to show a difference between the 2 groups. CONCLUSIONS: Riluzole appears to be safe in young children. None showed any change in motor abilities. None of the 10 subjects experienced adverse effects or changes in laboratory test results. Three of 7 who received active drug are still living at ages 513 years, 4 years, and 30 months. All 3 patients in the placebo group died (mean age, 9 months). RESULTS: Seven patients received riluzole and 3 received placebo medication. The investigational review boards of the participating centers approved the protocol and consent forms. The enrollment goal was 30 patients however, support from the pharmaceutical company was withdrawn when Rhone-Poulenc Rorer was taken over by Aventis. Treatment was reinstituted at 1 year if requested by the parents. Treatment was stopped after 9 months, and blood work was repeated at 12 months. An electrocardiogram was done at baseline, 3 months, 6 months, and 12 months. Complete blood count, hepatic and renal screens, and urinalysis were performed at baseline, 2 weeks, 1 month, 2 months, 3 months, 6 months, and 9 months after drug or placebo was started. Neurologic examination was performed at the first visit by one of the investigators. DESIGN: Subjects with homozygous deletions of the survival motor neuron gene were recruited from pediatric neuromuscular clinics and randomized in a 2:1 ratio, 2 riluzole to 1 placebo. OBJECTIVES: To determine whether a glutamate inhibitor might be tolerated by infants with SMA and, furthermore, whether this medication could have a positive effect on life expectancy. Riluzole, a glutamate inhibitor, has been shown to slow the rate of decline in patients with amyotrophic lateral sclerosis, another form of motor neuron disease. There is reason to believe that glutamate, an excitatory neurotransmitter, enhances programmed cell death of anterior horn cells. There is no known treatment for SMA, and, until recently, no therapeutic trials have been attempted. Severe spinal muscular atrophy (SMA) (Werdnig-Hoffmann disease, acute SMA, and SMA I) is a disease of the motor neuron characterized by onset before 6 months of age, failure ever to achieve sitting without support, and a life expectancy of 2 years or less. Oregon Health Sciences University and Shriners Hospital for Children, Portland, OR 97201, USA. #Grupo kairon trial#Como apenas 10 crianças foram incluídas para participar, sendo 7 usando a medicação e 3 usando placebo, o estudo não foi capaz de avaliar o efeito da droga sobre a doença, mas observou-se que não houve efeitos colaterais.Ī phase 1 trial of riluzole in spinal muscular atrophy. Neste estudo, pretendia-se avaliar o efeito de uma medicação inibidora de uma substância chamada glutamato, o riluzole, em 30 crianças com AME tipo 1. ![]()
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