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WAFMR, WSCI, WAP and WSPR Joint Plenary Session I 8:30 AM Thursday, January 26, 2012
1 A DOUBLE BLIND, PLACEBO CONTROLLED, CROSSOVER TRIAL OF MINOCYCLINE IN CHILDREN WITH FRAGILE X SYNDROME
Leigh M1 Nguyen D2, Winarni T3, Hessl D4, Rivera S5, Chechi T1, Hagerman R1 1MIND Institute, University of California Davis, Sacramento, CA; 2University of California Davis, Davis, CA; 3CEBIOR, Diponegoro University, Semarang, Indonesia; 4MIND Institute, University of California Davis, Sacramento, CA and 5Center for Mind and Brain, University of California Davis, Davis, CA.
Purpose of Study: The purpose of this study was to determine the efficacy and tolerability of minocycline as a targeted treatment for children with fragile X syndrome (FXS). Minocycline decreases matrix metalloproteinase 9 levels and rescues dendritic spine abnormalities in the fragile X knock out mouse. Prior open label human studies suggest benefits.
Methods Used: Children with FXS ages 3.5–16 years of age were randomized to receive minocycline or placebo. After three months, participants were crossed over to minocycline or placebo as appropriate for the following three months. Investigators and participants were blinded to the randomization. Outcome measures including the Clinical Global Impressions-Improvement (CGI-I) Scale, Visual Analogue Scale (VAS) for behaviors, and the Aberrant Behavior Checklist (ABC) were administered at baseline, 3 months and 6 months.
Summary of Results: This preliminary analysis focuses on 40 individuals, mean age 8.64 +/− 3.46 years. There was a significantly greater improvement in CGI-I scores with minocycline treatment compared to placebo (p=0.0274). The VAS showed a trend for greater improvement on minocycline when compared to placebo for the three behaviors identified by caregivers (p=0.2875, p=0.1296, 0.0551). The ABC Composite score also showed a trend towards greater improvement on minocycline (p=0.0628) when compared to placebo. No serious adverse events occurred and there was no significant difference in side effects during the minocycline period when compared to the placebo period.
Conclusions: Preliminary analysis supports …
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