An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

August 16, 2023 updated by: Sarepta Therapeutics, Inc.

Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

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).

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

171

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium, 3000
        • UZ Leuven
    • Oost-Vlaanderen
      • Gent, Oost-Vlaanderen, Belgium, 9000
        • UZ Gent
  • Bulgaria
      • Sofia, Bulgaria, 1431
        • University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B6A8
        • Alberta Children's Hospital
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L1
        • Children's Hospital of Eastern Ontario
  • Czechia
      • Brno, Czechia, 613 00
        • Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno
      • Praha 5, Czechia, 150 06
        • Fakultni nemocnice v Motole
  • France
      • Nantes Cedex, France, 44093
        • Hotel Dieu- CHU Nantes
      • Paris, France, 75013
        • Hôpital Armand Trousseau
  • Germany
    • Baden-Wurttemberg
      • Freiburg, Baden-Wurttemberg, Germany, 70106
        • Universitatsklinikum Freiberg
    • Nordrhein-Westfalen
      • Essen, Nordrhein-Westfalen, Germany, 45147
        • Universitätsklinikum Essen
  • Israel
      • Petach Tikva, Israel, 4920235
        • Schneider Children's Medical Center Of Israel
  • Italy
      • Cona, Italy, 44124
        • Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
      • Milano, Italy, 20123
        • Fondazione Policlinico Universitario A Gemelli
    • Liguria
      • Genova, Liguria, Italy, 16147
        • UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
  • Poland
      • Gdansk, Poland, 80-952
        • Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
    • Mazowieckie
      • Warsaw, Mazowieckie, Poland, 02-097
        • Samodzielny Publiczny Centralny Szpital Kliniczny
  • Spain
      • Barcelona, Spain, 8950
        • Hospital Sant Joan de Déu
      • Valencia, Spain, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia
    • Cataluña
      • Barcelona, Cataluña, Spain, 08041
        • Hospital de la Santa Creu i Sant Pau
  • Sweden
      • Goteborg, Sweden, 41685
        • Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust
      • Liverpool, United Kingdom, L12 2AP
        • Alder Hey Children's NHS Foundation Trust
      • London, United Kingdom, WC1N 1EH
        • Great Ormond Street Hospital (GOSH)
      • Newcastle Upon Tyne, United Kingdom, NE1 4LP
        • Royal Victoria Infirmary
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85028
        • Neuromuscular Research Center
    • California
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford Neuroscience Health Center
      • San Diego, California, United States, 92123
        • Rady Children's Hospital- San Diego
    • Florida
      • Gainesville, Florida, United States, 32610
        • UF Health: University of Florida Clinical Research Center
      • Gulf Breeze, Florida, United States, 32561
        • NW Florida Clinical Research Group, LLC
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Rare Disease Research, LLC
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann and Robert H Lurie Childrens Hospital of Chicago
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Childrens Hospital
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • St. Louis Children's Hospital
    • Nevada
      • Las Vegas, Nevada, United States, 89145
        • Las Vegas Clinic
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center - Department of Neurology
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Columbus, Ohio, United States, 43205
        • Abigail Wexner Research Institute at Nationwide Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Shriners Hospital for Children
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • Texas
      • Dallas, Texas, United States, 75207
        • Children's Medical Center Dallas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah - PPDS
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • Children's Hospital of The King's Daughters
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Children's Hopsital of Wisconsin, Corporate Center Suite 540

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Casimersen
Casimersen solution for IV infusion
Other Names:
  • SRP-4045
  • AMONDYS 45
Experimental: 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.
Drug: Golodirsen
Golodirsen solution for IV infusion
Other Names:
  • SRP-4053
  • VYONDYS 53

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Patients With Serious Adverse Events (SAEs)
Time Frame: Up to 30 days after the last infusion of study drug (assessed up to 148 weeks)
Up to 30 days after the last infusion of study drug (assessed up to 148 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 2, 2018

Primary Completion (Actual)

July 26, 2023

Study Completion (Actual)

July 26, 2023

Study Registration Dates

First Submitted

May 10, 2018

First Submitted That Met QC Criteria

May 10, 2018

First Posted (Actual)

May 22, 2018

Study Record Updates

Last Update Posted (Actual)

August 21, 2023

Last Update Submitted That Met QC Criteria

August 16, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 4045-302
  • 2017-004625-32 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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