An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
NCT ID: NCT03532542
Last Updated: 2024-09-19
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
171 participants
INTERVENTIONAL
2018-08-02
2023-07-26
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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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.
Casimersen
Casimersen solution for IV infusion
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.
Golodirsen
Golodirsen solution for IV infusion
Interventions
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Casimersen
Casimersen solution for IV infusion
Golodirsen
Golodirsen solution for IV infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Is between 7 and 23 years of age, inclusive, at enrollment.
7 Years
23 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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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
St 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
Children's Hospital of Wisconsin, Corporate Center Suite 540
Milwaukee, Wisconsin, United States
UZ Gent
Ghent, Oost-Vlaanderen, Belgium
UZ Leuven
Leuven, , 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
Prague, , Czechia
Hotel Dieu- CHU Nantes
Nantes, , France
Hôpital Armand Trousseau
Paris, , France
Universitatsklinikum Freiberg
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Universitatsklinikum Essen
Essen, North Rhine-Westphalia, Germany
Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
Genoa, Liguria, Italy
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Cona, , Italy
Fondazione Policlinico Universitario A Gemelli
Milan, , Italy
Samodzielny Publiczny Centralny Szpital Kliniczny
Warsaw, Masovian Voivodeship, Poland
Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
Gdansk, , Poland
Hospital de La Santa Creu i Sant Pau
Barcelona, Catalonia, Spain
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, , Spain
Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
Gothenburg, , 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
Countries
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Provided Documents
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Document Type: Study Protocol
Other Identifiers
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2017-004625-32
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
4045-302
Identifier Type: -
Identifier Source: org_study_id
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