An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
- Registration Number
- NCT03532542
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 171
- Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
- Is between 7 and 23 years of age, inclusive, at enrollment.
Other inclusion/exclusion criteria apply.
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Casimersen Casimersen Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. Golodirsen Golodirsen Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) Up to 33 days after the last infusion of study drug (up to approximately 149 weeks) A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
- Secondary Outcome Measures
Name Time Method
Related Research Topics
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Trial Locations
- Locations (50)
Fondazione Policlinico Universitario A Gemelli
🇮🇹Milano, Italy
UZ Leuven
🇧🇪Leuven, Belgium
Children's Hospital of Wisconsin, Corporate Center Suite 540
🇺🇸Milwaukee, Wisconsin, United States
Neuromuscular Research Center
🇺🇸Phoenix, Arizona, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
University of California Los Angeles
🇺🇸Los Angeles, California, United States
Stanford Neuroscience Health Center
🇺🇸Palo Alto, California, United States
Rady Children's Hospital- San Diego
🇺🇸San Diego, California, United States
UF Health: University of Florida Clinical Research Center
🇺🇸Gainesville, Florida, United States
NW Florida Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Iowa Childrens Hospital
🇺🇸Iowa City, Iowa, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Las Vegas Clinic
🇺🇸Las Vegas, Nevada, United States
University of Rochester Medical Center - Department of Neurology
🇺🇸Rochester, New York, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Abigail Wexner Research Institute at Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Shriners Hospital for Children
🇺🇸Portland, Oregon, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Children's Medical Center Dallas
🇺🇸Dallas, Texas, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
University of Utah - PPDS
🇺🇸Salt Lake City, Utah, United States
Children's Hospital of The King's Daughters
🇺🇸Norfolk, Virginia, United States
UZ Gent
🇧🇪Gent, Oost-Vlaanderen, Belgium
University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases
🇧🇬Sofia, Bulgaria
Alberta Children's Hospital
🇨🇦Calgary, Alberta, Canada
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno
🇨🇿Brno, Czechia
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
Hotel Dieu- CHU Nantes
🇫🇷Nantes Cedex, France
Hôpital Armand Trousseau
🇫🇷Paris, France
Universitatsklinikum Freiberg
🇩🇪Freiburg, Baden-Wurttemberg, Germany
Universitatsklinikum Essen
🇩🇪Essen, Nordrhein-Westfalen, Germany
Schneider Children's Medical Center of Israel
🇮🇱Petach Tikva, Israel
UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
🇮🇹Genova, Liguria, Italy
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
🇮🇹Cona, Italy
Samodzielny Publiczny Centralny Szpital Kliniczny
🇵🇱Warsaw, Mazowieckie, Poland
Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
🇵🇱Gdansk, Poland
Hospital de La Santa Creu i Sant Pau
🇪🇸Barcelona, Cataluña, Spain
Hospital Sant Joan de Deu
🇪🇸Barcelona, Spain
Hospital Universitari i Politecnic La Fe de Valencia
🇪🇸Valencia, Spain
Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
🇸🇪Goteborg, Sweden
Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust
🇬🇧Leeds, United Kingdom
Alder Hey Children's NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
Great Ormond Street Hospital (GOSH)
🇬🇧London, United Kingdom
Royal Victoria Infirmary
🇬🇧Newcastle Upon Tyne, United Kingdom