Ignite Creation Date:
2024-05-06 @ 2:47 AM
Last Modification Date:
2024-10-26 @ 11:23 AM
Study NCT ID:
NCT02126995
Status:
COMPLETED
Last Update Posted:
2016-07-06
First Post:
2014-04-29
Brief Title:
A 6-week Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Sponsor:
Alcobra Ltd
Organization:
Alcobra Ltd
Study Overview
Official Title:
A 6-week Randomized Multicenter Double-blind Parallel Flexed and Fixed-dose Study of MG01CI Metadoxine Extended-release Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Status:
COMPLETED
Status Verified Date:
2014-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 11 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 visitsphone assessments the investigator will evaluate the dose based upon the investigators 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
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 visitsphone assessments the investigator will evaluate the dose based upon the investigators 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 11 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 subjects 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 visitsphone assessments the investigator will evaluate the dose based upon the investigators 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
The first 4 weeks of the treatment period will be a dose-optimization period during which the subjects 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 visitsphone assessments the investigator will evaluate the dose based upon the investigators 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None