A Randomized Double-Blind, Placebo-Controlled Trial of Minocycline in Children and Adolescents with Fragile X Syndrome

Leight, Mary Jacena S. and Nguyen, Danh V. and Yi Mu, Yi Mu and Winarni, Tri Indah and Schneider, Andrea and Chechi, Tasleem and Polussa, Jonathan and Doucet, Paul and Tassone, Flora and Rivera, Susan M. (2013) A Randomized Double-Blind, Placebo-Controlled Trial of Minocycline in Children and Adolescents with Fragile X Syndrome. Journal of Development and Behavioral Pediatrics, 34 (3). pp. 147-155. ISSN 1536-7312

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Official URL: https://journals.lww.com/jrnldbp/pages/currenttoc....


Objective: Minocycline rescued synaptic abnormalities and improved behavior in the fragile X mouse model. Previous open-label human studies demonstrated benefits in individuals with fragile X syndrome (FXS); however, its efficacy in patients with FXS has not been assessed in a controlled trial. Method: Randomized, double-blind, placebo-controlled, crossover trial in individuals with FXS, aged 3.5 years to 16 years (n 5 55, mean age 9.2 [SD, 3.6] years). Participants were randomized to minocycline or placebo for 3 months and then switched to the other treatment. Results: Sixty-nine subjects were screened and 66 were randomized. Fifty-five subjects (83.3%) completed at least the first period and 48 (72.7%) completed the full trial. Intention-to-treat analysis demonstrated significantly greater improvements in one primary outcome, Clinical Global Impression Scale—Improvement after minocycline compared with placebo (2.49 6 0.13 and 2.97 6 0.13, respectively, p 5 .0173) and greater improvement in ad hoc analysis of anxiety and mood-related behaviors on the Visual Analog Scale (minocycline: 5.26 cm 6 0.46 cm, placebo: 4.05 cm 6 0.46 cm; p 5 .0488). Side effects were not significantly different during the minocycline and placebo treatments. No serious adverse events occurred on minocycline. Results may be potentially biased by study design weaknesses, including unblinding of subjects when they completed the study, drug-related side effects unblinding, and preliminary efficacy analysis results known to investigators. Conclusions: Minocycline treatment for 3 months in children with FXS resulted in greater global improvement than placebo. Treatment for 3 months appears safe; however, longer trials are indicated to further assess benefits, side effects, and factors associated with a clinical response to minocycline.

Item Type:Article
Subjects:R Medicine > R Medicine (General)
Divisions:Faculty of Medicine > Department of Medicine
Faculty of Medicine > Department of Medicine
ID Code:67599
Deposited By:Ms Lutfiatun Heni
Deposited On:07 Dec 2018 11:04
Last Modified:07 Dec 2018 11:04

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