Ignite Creation Date:
2024-05-06 @ 7:42 PM
Last Modification Date:
2024-10-26 @ 3:12 PM
Study NCT ID:
NCT06100887
Status:
RECRUITING
Last Update Posted:
2024-03-27
First Post:
2023-10-20
Brief Title:
Phase 2 Study of EDG-5506 in Children and Adolescents with Duchenne Muscular Dystrophy Previously Treated with Gene Therapy FOX
Sponsor:
Edgewise Therapeutics Inc
Organization:
Edgewise Therapeutics Inc
Study Overview
Official Title:
A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety Pharmacokinetics and Biomarkers in Children and Adolescents with Duchenne Muscular Dystrophy Previously Treated with Gene Therapy
Status:
RECRUITING
Status Verified Date:
2024-09
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:
The FOX study is a 2-part multicenter Phase 2 study of safety pharmacokinetics and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized double-blind placebo-controlled Part A followed by an open-label part B
Detailed Description:
The EDG-5506-215 protocol was amended to include an additional dose cohort and enroll additional participants across select cohorts
FOX is a 2-part multi-center Phase 2 study to evaluate the effect of sevasemten EDG-5506 on safety pharmacokinetics and biomarkers of muscle damage in approximately 48 children and adolescents with Duchenne muscular dystrophy treated with oral once-daily sevasemten This study will have up to a 4-week Screening period a 12-week randomized double-blind placebo-controlled treatment period Part A followed by a 40-week open-label extension period Part B
Approximately forty-eight 48 participants aged 6 to 17 inclusive will be randomized to sevasemten or placebo in a 21 ratio Three dose cohorts Cohort 1 Cohort 2 and Cohort 3 of approximately 12 participants each will be enrolled Approximately 12 additional participants may be added to 1 of these cohorts following Data Monitoring Committee DMC review of emerging data
FOX is a 2-part multi-center Phase 2 study to evaluate the effect of sevasemten EDG-5506 on safety pharmacokinetics and biomarkers of muscle damage in approximately 48 children and adolescents with Duchenne muscular dystrophy treated with oral once-daily sevasemten This study will have up to a 4-week Screening period a 12-week randomized double-blind placebo-controlled treatment period Part A followed by a 40-week open-label extension period Part B
Approximately forty-eight 48 participants aged 6 to 17 inclusive will be randomized to sevasemten or placebo in a 21 ratio Three dose cohorts Cohort 1 Cohort 2 and Cohort 3 of approximately 12 participants each will be enrolled Approximately 12 additional participants may be added to 1 of these cohorts following Data Monitoring Committee DMC review of emerging data
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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