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document
Effect of CX516 , an AMPA-modulating compound , on cognition and behavior in fragile X syndrome : a controlled trial . A Phase II , 4-week randomized , double-blind , placebo-controlled clinical trial was conducted to evaluate the safety and efficacy of the Ampakine compound CX516 as a potential treatment for the underlying disorder in fragile X syndrome ( FXS ) . After baseline screening , subjects with FXS ( n = 49 ) underwent a 1-week placebo lead-in and then were randomized to study drug or placebo for a 4-week period . Cognitive and behavioral outcome measures were administered prior to treatment , at the end of treatment , and 2 weeks posttreatment . There were minimal side effects , no significant changes in safety parameters , and no serious adverse events . There was a 12.5 % frequency of allergic rash in the CX516 group and 1 subject developed a substantial rash . There was also no significant improvement in memory , the primary outcome measure , or in secondary measures of language , attention/executive function , behavior , and overall functioning in CX516-treated subjects compared to placebo . This study did demonstrate that many outcome measures were reproducible in this test-retest setting for the FXS population , yet some were too difficult or variable . Adult subjects with FXS were able to complete an intensive clinical trial , and some valid outcome measures were identified for future FXS trial design . Problems with potency of CX516 in other studies have suggested dosing may have been inadequate for therapeutic effect and thus it remains unclear whether modulation of AMPA-mediated neurotransmission is a viable therapeutic strategy for the treatment of FXS .
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Intervention_Pharmacological
CX516
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Intervention_Pharmacological
AMPA-modulating compound
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Intervention_Control
placebo-controlled
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Intervention_Pharmacological
Ampakine
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Intervention_Pharmacological
CX516
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Intervention_Control
placebo
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Intervention_Control
placebo
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Intervention_Pharmacological
CX516
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Intervention_Pharmacological
CX516-treated
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Intervention_Control
placebo
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Intervention_Pharmacological
CX516
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Outcome_Mental
cognition
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Outcome_Physical
and
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Outcome_Mental
behavior
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Outcome_Other
safety and efficacy
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Outcome_Mental
Cognitive and behavioral
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Outcome_Adverse-effects
minimal side effects
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Outcome_Other
significant changes in safety parameters
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Outcome_Adverse-effects
no serious adverse events .
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Outcome_Adverse-effects
12.5 % frequency
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Outcome_Adverse-effects
allergic rash
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Outcome_Adverse-effects
substantial rash
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Outcome_Mental
significant improvement
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Outcome_Mental
memory
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Outcome_Mental
language , attention/executive function , behavior , and overall functioning
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Outcome_Other
measures
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Outcome_Other
viable therapeutic strategy
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Participant_Condition
cognition and behavior in fragile X syndrome
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Participant_Condition
underlying disorder in fragile X syndrome ( FXS
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Participant_Sample-size
n = 49
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Participant_Age
Adult subjects
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Participant_Condition
FXS