Ignite Creation Date:
2024-05-06 @ 2:21 AM
Last Modification Date:
2024-10-26 @ 11:17 AM
Study NCT ID:
NCT02023424
Status:
UNKNOWN
Last Update Posted:
2014-02-04
First Post:
2013-12-23
Brief Title:
An Open Label Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome
Sponsor:
Sheba Medical Center
Organization:
Sheba Medical Center
Study Overview
Official Title:
An Open Label Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate Copaxone on Girls Woth Rett Syndrome
Status:
UNKNOWN
Status Verified Date:
2014-02
Last Known Status:
RECRUITING
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:
Primary Objective To test the hypothesis that 6 months treatment with glatiramer acetate GA decreases epileptiform activity in young girls with Rett syndrome
Primary Safety ObjectiveTo evaluate the safety and tolerability of 6 months treatment with GA in these patients
Secondary Objectives
1 To test the hypothesis that 6 months treatment with glatiramer acetate GA improves respiratory dysfunction
2 To evaluate the effect of GA treatment on general behaviour communication hand stereotyping feeding sleep and other autonomic symptoms gastrointestinal and cardiac
3 To assess the effect of GA treatment on bodily development
Primary EndpointImprovement of epileptiform activity as recorded in a 24-hours EEG
Primary Safety EndpointFrequency and severity of treatment-related AEs including safety lab parameters
Secondary Endpoints
1 Improvement in the scoring of breath holds and hyperventilation as measured with non-invasive respiratory inductance plethysmography NoxT3 device and parents diaries
2 Changes in general behaviour communication feeding and motor skills as assessed by the investigator based on Kerr and Naidu validated severity scores and recorded in parents diary
3 Decrease in seizure frequency as reported in parents diary
4 Improvement in sleep schedule as recorded in a sleep diary
5 Change in height and weight PopulationTen girls 6 to 15 years old diagnosed with Rett syndrome RTT Study DesignThis is a single - center exploratory open-label study in 10 girls diagnosed with RTT The study will consist of four parts Screening and baseline assessments initial and final dose-setting period treatment period and end-of study follow-up
Investigational ProductGlatiramer Acetate Copaxone Teva Pharmaceutical Industries Ltd Sample Size Consideration The planned sample size of 10 patients was considered adequate by the investigator for this phase I exploratory proof-of-concept study The study is not expected to show statistical significance or statistical power only a trend for the study endpoints Each patient will serve as her own control
Duration of Study Approximately 8 months per patient including up to 2 weeks pre-treatment assessment 6 months initial dose and treatment periods and end-of study visit
Overall study duration the study is expected to be completed within 12 months dependent on rate of recruitment
Primary Safety ObjectiveTo evaluate the safety and tolerability of 6 months treatment with GA in these patients
Secondary Objectives
1 To test the hypothesis that 6 months treatment with glatiramer acetate GA improves respiratory dysfunction
2 To evaluate the effect of GA treatment on general behaviour communication hand stereotyping feeding sleep and other autonomic symptoms gastrointestinal and cardiac
3 To assess the effect of GA treatment on bodily development
Primary EndpointImprovement of epileptiform activity as recorded in a 24-hours EEG
Primary Safety EndpointFrequency and severity of treatment-related AEs including safety lab parameters
Secondary Endpoints
1 Improvement in the scoring of breath holds and hyperventilation as measured with non-invasive respiratory inductance plethysmography NoxT3 device and parents diaries
2 Changes in general behaviour communication feeding and motor skills as assessed by the investigator based on Kerr and Naidu validated severity scores and recorded in parents diary
3 Decrease in seizure frequency as reported in parents diary
4 Improvement in sleep schedule as recorded in a sleep diary
5 Change in height and weight PopulationTen girls 6 to 15 years old diagnosed with Rett syndrome RTT Study DesignThis is a single - center exploratory open-label study in 10 girls diagnosed with RTT The study will consist of four parts Screening and baseline assessments initial and final dose-setting period treatment period and end-of study follow-up
Investigational ProductGlatiramer Acetate Copaxone Teva Pharmaceutical Industries Ltd Sample Size Consideration The planned sample size of 10 patients was considered adequate by the investigator for this phase I exploratory proof-of-concept study The study is not expected to show statistical significance or statistical power only a trend for the study endpoints Each patient will serve as her own control
Duration of Study Approximately 8 months per patient including up to 2 weeks pre-treatment assessment 6 months initial dose and treatment periods and end-of study visit
Overall study duration the study is expected to be completed within 12 months dependent on rate of recruitment
Detailed Description:
None
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