Duchenne Muscular Dystrophy Clinical Trial
Official title:
Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
| Verified date | August 2023 |
| Source | Sarepta Therapeutics, Inc. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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).
| Status | Terminated |
| Enrollment | 171 |
| Est. completion date | July 26, 2023 |
| Est. primary completion date | July 26, 2023 |
| Accepts healthy volunteers | No |
| Gender | Male |
| Age group | 7 Years to 23 Years |
| Eligibility | Inclusion Criteria: - 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. |
| Country | Name | City | State |
|---|---|---|---|
| Belgium | UZ Gent | Gent | Oost-Vlaanderen |
| Belgium | UZ Leuven | Leuven | |
| Bulgaria | University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases | Sofia | |
| Canada | Alberta Children's Hospital | Calgary | Alberta |
| Canada | Children's Hospital of Eastern Ontario | Ottawa | Ontario |
| Czechia | Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno | Brno | |
| Czechia | Fakultni nemocnice v Motole | Praha 5 | |
| France | Hotel Dieu- CHU Nantes | Nantes Cedex | |
| France | Hôpital Armand Trousseau | Paris | |
| Germany | Universitatsklinikum Essen | Essen | Nordrhein-Westfalen |
| Germany | Universitatsklinikum Freiberg | Freiburg | Baden-Wurttemberg |
| Israel | Schneider Children's Medical Center of Israel | Petach Tikva | |
| Italy | Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna | Cona | |
| Italy | UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative | Genova | Liguria |
| Italy | Fondazione Policlinico Universitario A Gemelli | Milano | |
| Poland | Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej | Gdansk | |
| Poland | Samodzielny Publiczny Centralny Szpital Kliniczny | Warsaw | Mazowieckie |
| Spain | 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 | |
| Sweden | Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus | Goteborg | |
| United Kingdom | 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 States | Rare Disease Research, LLC | Atlanta | Georgia |
| United States | Kennedy Krieger Institute | Baltimore | Maryland |
| United States | Boston Children's Hospital | Boston | Massachusetts |
| United States | Ann and Robert H Lurie Childrens Hospital of Chicago | Chicago | Illinois |
| 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 | Children's Medical Center Dallas | Dallas | Texas |
| 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 | Texas Children's Hospital | Houston | Texas |
| United States | University of Iowa Childrens Hospital | Iowa City | Iowa |
| United States | University of Kansas Medical Center | Kansas City | Kansas |
| United States | Las Vegas Clinic | Las Vegas | Nevada |
| United States | Children's Hospital Los Angeles | Los Angeles | California |
| United States | University of California Los Angeles | Los Angeles | California |
| United States | Children's Hopsital of Wisconsin, Corporate Center Suite 540 | Milwaukee | Wisconsin |
| United States | Children's Hospital of The King's Daughters | Norfolk | Virginia |
| United States | Stanford Neuroscience Health Center | Palo Alto | California |
| United States | Neuromuscular Research Center | Phoenix | Arizona |
| United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
| United States | Shriners Hospital for Children | Portland | Oregon |
| United States | University of Rochester Medical Center - Department of Neurology | Rochester | New York |
| United States | St. Louis Children's Hospital | Saint Louis | Missouri |
| United States | University of Utah - PPDS | Salt Lake City | Utah |
| United States | Rady Children's Hospital- San Diego | San Diego | California |
| Lead Sponsor | Collaborator |
|---|---|
| Sarepta Therapeutics, Inc. |
United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, Italy, Poland, Spain, Sweden, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Patients With Serious Adverse Events (SAEs) | Up to 30 days after the last infusion of study drug (assessed up to 148 weeks) |
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