Clinical trial NCT03532542: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Official title: Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne muscular dystrophy
Exon Skipping
DMD
Exon 53
Exon 45
Duchenne
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-05-22
Enrollment: 171
Sex: MALE
Minimum age (years): 7
Maximum age (years): 23
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
timeFrame: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Neuromuscular Research Center
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford Neuroscience Health Center
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital- San Diego
state: California
city: Gainesville
country: United States
facility: UF Health: University of Florida Clinical Research Center
state: Florida
city: Gulf Breeze
country: United States
facility: NW Florida Clinical Research Group, LLC
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H Lurie Childrens Hospital of Chicago
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Childrens Hospital
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Las Vegas
country: United States
facility: Las Vegas Clinic
state: Nevada
city: Rochester
country: United States
facility: University of Rochester Medical Center - Department of Neurology
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Abigail Wexner Research Institute at Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Shriners Hospital for Children
state: Oregon
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Dallas
country: United States
facility: Children's Medical Center Dallas
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah - PPDS
state: Utah
city: Norfolk
country: United States
facility: Children's Hospital of The King's Daughters
state: Virginia
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin, Corporate Center Suite 540
state: Wisconsin
city: Gent
country: Belgium
facility: UZ Gent
state: Oost-Vlaanderen
city: Leuven
country: Belgium
facility: UZ Leuven
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole
city: Nantes Cedex
country: France
facility: Hotel Dieu- CHU Nantes
city: Paris
country: France
facility: Hôpital Armand Trousseau
city: Freiburg
country: Germany
facility: Universitatsklinikum Freiberg
state: Baden-Wurttemberg
city: Essen
country: Germany
facility: Universitatsklinikum Essen
state: Nordrhein-Westfalen
city: Petach Tikva
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Genova
country: Italy
facility: UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
state: Liguria
city: Cona
country: Italy
facility: Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
city: Milano
country: Italy
facility: Fondazione Policlinico Universitario A Gemelli
city: Warsaw
country: Poland
facility: Samodzielny Publiczny Centralny Szpital Kliniczny
state: Mazowieckie
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
city: Barcelona
country: Spain
facility: Hospital de La Santa Creu i Sant Pau
state: Cataluña
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
city: Goteborg
country: Sweden
facility: Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital (GOSH)
city: Newcastle Upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
Study officials:
affiliation: Sarepta Therapeutics, Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: