A Randomized Double-Blind, Placebo-Controlled Trial ofMinocycline in Children and Adolescents with Fragile XSyndrome
2013
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.
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