Duchenne Muscular Dystrophy (DMD) Clinical Trial
Official title:
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.
This is an open label, multiple dose extension study to assess the ongoing efficacy, safety,
and tolerability of weekly intravenous (IV) infusions of eteplirsen in DMD subjects who have
successfully completed Study 4658-us 201.
Subjects will have the opportunity to enroll in this study during the last visit of Study
4658-us-201 (Week 28). Eligible subjects will receive once weekly IV infusions of eteplirsen
(50 or 30 mg/kg) for an additional 212 weeks. Subjects will receive the same dose of
eteplirsen they received in Study 4658-us-201. Subjects will thereafter continue to receive
once weekly IV infusions of eteplirsen for up to an additional 72 week period (through week
284). If commercial eteplirsen becomes available during this additional 72 week period,
participation in the study will be discontinued as subjects transition to commercial
eteplirsen.
Safety, efficacy, pharmacokinetic (PK), and biomarker assessments will be performed at
scheduled visits; adverse events (AEs) and concomitant medications and therapies will be
continuously monitored.
If review of data from this open label study suggests that continued treatment with
eteplirsen is warranted, this study may be extended by protocol amendment or subjects who
successfully complete this study may have the opportunity to participate in a separate follow
on, open label eteplirsen study.
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT02814019 -
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
|
Phase 3 | |
| Completed |
NCT01478022 -
To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
|
Phase 1 | |
| Completed |
NCT00654784 -
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
|
Phase 2 | |
| Recruiting |
NCT02614820 -
The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD
|
N/A | |
| Withdrawn |
NCT02036463 -
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
|
Phase 2 | |
| Completed |
NCT02255552 -
Study of Eteplirsen in DMD Patients
|
Phase 3 | |
| Completed |
NCT01982695 -
Cardiomyopathy in DMD: Lisinopril vs. Losartan
|
N/A | |
| Recruiting |
NCT05516745 -
E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"
|
N/A | |
| Completed |
NCT02420379 -
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
|
Phase 2 | |
| Active, not recruiting |
NCT05135663 -
Extension Study of NS-089/NCNP-02 in DMD
|
Phase 2 | |
| Completed |
NCT01380964 -
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
|
N/A | |
| Completed |
NCT05990608 -
Peabody Developmental Motor Scaling In Children With Period Duchenne Muscular Dystrophy
|
||
| Recruiting |
NCT04322357 -
Twice Weekly Steroids and Exercise as Therapy for DMD
|
Phase 2 | |
| Not yet recruiting |
NCT06392724 -
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.
|
Early Phase 1 | |
| Terminated |
NCT04184882 -
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 1 | |
| Terminated |
NCT04254172 -
A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy
|
||
| Recruiting |
NCT05524883 -
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
|
Phase 1/Phase 2 | |
| Completed |
NCT01761292 -
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
|
Phase 1/Phase 2 |