A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Condition: Fragile X Syndrome
MeSH terms: Fragile X Syndrome; Syndrome
Study type: Interventional
Phase: Phase 2

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.
    

Detailed description: 
      This study is a multisite, randomized, double-blind, placebo-controlled, phase 2 study of
      MG01CI (low and high doses of metadoxine once daily) for 6 weeks compared with placebo in a
      1:1 ratio of 60 adolescent and adult subjects with 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, during which
      the subject's dose of MG01CI or placebo will be optimized. Investigators and subjects will be
      blinded with regard to whether the subject is taking active drug or placebo. Subjects will be
      blinded to anticipated dose (low vs high dose), while Investigators will not be blinded to
      anticipated dose, low dose vs high dose. Every two weeks subjects will receive 2-week supply
      treatment. A phone follow-up assessment of safety and tolerability will occur during
      titration after 1 and 3 weeks of treatment; if the investigator has any significant concerns
      regarding safety and tolerability, the subject will be assessed at the site at an unscheduled
      visit. All subjects will be assessed at the site after 2 weeks and 4 weeks of treatment.

      All subjects will start with either low dose or matching blinded placebo (2 tablets daily).
      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 2 tablets of either high dose of active
      treatment or placebo. If at 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.

      The last 2 weeks of the treatment period will be a dose-maintenance period. During the
      dose-maintenance period, the subject will maintain his or her optimal dose as determined at
      the end of the dose-optimization period. A phone follow-up assessment of safety and
      tolerability will occur after 5 weeks of treatment (after 1 week of dose maintenance). If the
      investigator has any significant concerns regarding safety and tolerability, the subject will
      be assessed at the site at an unscheduled visit. The subject will be assessed at the site
      after 6 weeks of treatment (after 2 weeks of dose maintenance).

      There will be a 2-week Follow-up Period after the last dose of study treatment or early
      termination.
    

Intervention name(s): MG01CI extended-release tablet
Intervention type(s): Drug
Intervention description(s): Metadoxine, MG01CI extended-release tablet taken for 6 weeks treatment. Starting with a dose of 700 mg for 2 weeks followed by a 2 weeks of 1400 mg and then 2 weeks of 700 mg or 1400 mg.
Other intervention name(s): Metadoxine (pyridoxol L-2-pyrrolidone-5-carboxylate)
Intervention MeSH terms: Metadoxine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: Fragile X Syndrome, Fra(X) Syndrome, FRAXA Syndrome