Ignite Creation Date:
2024-05-06 @ 3:24 PM
Last Modification Date:
2024-10-26 @ 1:49 PM
Study NCT ID:
NCT04622553
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-06
First Post:
2020-10-22
Brief Title:
Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study
Sponsor:
Lupin Ltd
Organization:
Lupin Ltd
Study Overview
Official Title:
Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients With Myotonic Disorders Who Have Completed the MEX-NM-301 Study
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-05
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:
Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed the MEX-NM-301 study
Detailed Description:
This is an open-label extension study evaluating the long-term efficacy and safety of mexiletine in paediatric patients with myotonic disorders who have completed the initial parent paediatric study with mexiletine Protocol No MEX-NM-301 PIP Study 4 for children and adolescents aged 6 to 18 years and who continue to meet the eligibility criteria
Patients who meet the eligibility criteria and provide consent for this study will be enrolled sequentially by decreasing age groups Patients aged 12 to 18 years will enter first as this is the first cohort expected to complete the parent study PIP Study 4 based on top down recruiting Once initial pharmacokinetics PK safety and efficacy are confirmed in this population patients aged 6 to 12 years will be first enrolled in PIP Study 4 and subsequently this study PIP Study 7
Enrolled patients will receive mexiletine at a dose determined in the parent study Dosing is determined according to body weight and tolerability
The study includes 9 clinic visits - V1 baseline and V2 to V9 every 3 months approximately thereafter
The total duration of study will be 24 months per patient End-of-treatment EOT visit will occur at 24 months or in accordance with the availability of product The overall study duration would be approximately 5 years
Patients who meet the eligibility criteria and provide consent for this study will be enrolled sequentially by decreasing age groups Patients aged 12 to 18 years will enter first as this is the first cohort expected to complete the parent study PIP Study 4 based on top down recruiting Once initial pharmacokinetics PK safety and efficacy are confirmed in this population patients aged 6 to 12 years will be first enrolled in PIP Study 4 and subsequently this study PIP Study 7
Enrolled patients will receive mexiletine at a dose determined in the parent study Dosing is determined according to body weight and tolerability
The study includes 9 clinic visits - V1 baseline and V2 to V9 every 3 months approximately thereafter
The total duration of study will be 24 months per patient End-of-treatment EOT visit will occur at 24 months or in accordance with the availability of product The overall study duration would be approximately 5 years
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None