A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Status:
Completed
Trial end date:
2015-06-01
Target enrollment:
Participant gender:
Summary
This study is a multisite, randomized, double-blind, placebo-controlled, phase 2 study of
MG01CI (low dose and high dose once daily) for 6 weeks compared with placebo in a 1:1 ratio
of 60 adolescent and adult subjects with Fragile X Syndrome (FXS). Following Screening,
subjects will be randomized to MG01CI or matching placebo at Baseline (Day 0) and the 6 week
Double-blind Treatment Period will begin on Day 1.
The first 4 weeks of the treatment period will be a dose-optimization period,
All subjects will start with two daily tablets: low dose metadoxine or matching blinded
placebo. At weekly visits/phone assessments, the investigator will evaluate the dose based
upon the investigator's assessment of safety and tolerability. If the subject demonstrates
safety or tolerability concerns with the low dose after 1 or 2 weeks of treatment, then the
subject will be discontinued. If there are no concerns about safety and tolerability after 2
weeks of treatment, then the dose will be increased to high dose or placebo. If at the high
dose there are concerns about safety and tolerability, then the dose will be either kept the
same or reduced to low dose for the remainder of the treatment period.
There will be a 2-week Follow-up Period after the last dose of study treatment or early
termination.