Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06564974
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-09
Brief Title:
Registry Study to Observe Long-term Safety of Vamorolone AGAMREE in Patients with Duchenne Muscular Dystrophy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Registry Study to Observe Long-term Safety of Vamorolone AGAMREE in Patients with Duchenne Muscular Dystrophy
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE in male patients 2 years of age or older with Duchennes Muscular Dystrophy for long term safety and quality of life
Detailed Description:
This is a multi-center observational prospective longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE
This registry will be conducted in the US at approximately 25 sites known to treat and follow patients with DMD The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD
Evaluations will include
Growth parameters
Body mass index BMI
Vital Signs
Physical Exam
Laboratory Chemistry and Hematology
North Star Ambulatory Assessment NSAA
Performance of Upper Limb PUL
Cardiovascular status
Fractures
Bone density
Puberty
Quality of life QoL
Adverse events AEs
Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits - 30 days for registry assessments Information on standard of care treatment and procedures for management of DMD will also be collected Patients andor their parentslegal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit - 30 days
This registry will be conducted in the US at approximately 25 sites known to treat and follow patients with DMD The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD
Evaluations will include
Growth parameters
Body mass index BMI
Vital Signs
Physical Exam
Laboratory Chemistry and Hematology
North Star Ambulatory Assessment NSAA
Performance of Upper Limb PUL
Cardiovascular status
Fractures
Bone density
Puberty
Quality of life QoL
Adverse events AEs
Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits - 30 days for registry assessments Information on standard of care treatment and procedures for management of DMD will also be collected Patients andor their parentslegal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit - 30 days
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