Brief Summary
This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.
Brief Title
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Categories
Completion Date
Completion Date Type
Estimated
Conditions
Duchenne Muscular Dystrophy
Eligibility Criteria
Key Inclusion Criteria:
* Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
* Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
* Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
Key Exclusion Criteria:
* Is currently participating in any DMD interventional study at the time of this study enrollment.
* Has declined to provide consent for collection of their genetic data.
* Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:
1. The participant's ability to comply with the protocol-required procedures
2. The participant's wellbeing or safety, and/or
3. The clinical interpretability of the data collected from the participant.
Other inclusion/exclusion criteria may apply.
* Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
* Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
* Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
Key Exclusion Criteria:
* Is currently participating in any DMD interventional study at the time of this study enrollment.
* Has declined to provide consent for collection of their genetic data.
* Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:
1. The participant's ability to comply with the protocol-required procedures
2. The participant's wellbeing or safety, and/or
3. The clinical interpretability of the data collected from the participant.
Other inclusion/exclusion criteria may apply.
Inclusion Criteria
Inclusion Criteria:
* Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
* Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
* Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
inclusion/
* Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
* Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
* Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
inclusion/
Gender
Male
Gender Based
false
Keywords
Duchenne Muscular Dystrophy
DMD
Post-marketing
Observational
Phase 4
Eteplirsen
Golodirsen
Casimersen
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Estimated
Last Update Submit Date
NCT Id
NCT06606340
Org Class
Industry
Org Full Name
Sarepta Therapeutics, Inc.
Org Study Id
4658-403
Overall Status
Enrolling by invitation
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Primary Outcomes
Outcome Measure
Loss of Ambulation (LOA)
Outcome Time Frame
Up to 5 years
Outcome Measure
Time to Rise From the Floor (Supine to Stand)
Outcome Time Frame
Up to 5 years
Outcome Measure
Time to Walk/Run 10 Meters
Outcome Time Frame
Up to 5 years
Outcome Measure
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
Outcome Time Frame
Up to 5 years
Outcome Measure
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
Outcome Time Frame
Up to 5 years
Outcome Measure
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Outcome Time Frame
Up to 5 years
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Study Population
Participants within each prescriber's practice who are receiving eteplirsen, golodirsen, or casimersen, in routine clinical practice, and who meet the study eligibility criteria and provide informed consent (either by the participant or through authorization by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
LESLIE DELFINER
Investigator Email
ldelfine@montefiore.org
Investigator Phone
718-9250-4378
Investigator Department
Neurology
Investigator Division
Child Neurology
Investigator Sponsor Organization
Montefiore
Study Department
Please Specify
Study Division
Please Specify
Categories Mesh Debug
Arthritis --- MUSCULOSKELETAL DISEASES
Child Development & Autism --- MUSCULOSKELETAL DISEASES
Orthopedics, Muscle & Bone --- MUSCULOSKELETAL DISEASES
Brain, Spinal Cord & Nervous System --- NEUROMUSCULAR DISEASES
Brain, Spinal Cord & Nervous System --- NERVOUS SYSTEM DISEASES
Brain, Spine & Nerve Cancers --- NERVOUS SYSTEM DISEASES
MeSH Terms
MUSCULAR DYSTROPHY, DUCHENNE
MUSCULAR DYSTROPHIES
MUSCULAR DISORDERS, ATROPHIC
MUSCULAR DISEASES
MUSCULOSKELETAL DISEASES
NEUROMUSCULAR DISEASES
NERVOUS SYSTEM DISEASES
GENETIC DISEASES, X-LINKED
GENETIC DISEASES, INBORN
CONGENITAL, HEREDITARY, AND NEONATAL DISEASES AND ABNORMALITIES
ETEPLIRSEN
GOLODIRSEN
CASIMERSEN