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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Casimersen
Golodirsen
Sponsored by
Sarepta Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Duchenne

Eligibility Criteria

7 Years - 23 Years (Child, Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Neuromuscular Research Center
  • Children's Hospital Los Angeles
  • University of California Los Angeles
  • Stanford Neuroscience Health Center
  • Rady Children's Hospital- San Diego
  • UF Health: University of Florida Clinical Research Center
  • NW Florida Clinical Research Group, LLC
  • Rare Disease Research, LLC
  • Ann and Robert H Lurie Childrens Hospital of Chicago
  • University of Iowa Childrens Hospital
  • University of Kansas Medical Center
  • Kennedy Krieger Institute
  • Boston Children's Hospital
  • St. Louis Children's Hospital
  • Las Vegas Clinic
  • University of Rochester Medical Center - Department of Neurology
  • Cincinnati Children's Hospital Medical Center
  • Abigail Wexner Research Institute at Nationwide Children's Hospital
  • Shriners Hospital for Children
  • Children's Hospital of Pittsburgh of UPMC
  • Children's Medical Center Dallas
  • Texas Children's Hospital
  • University of Utah - PPDS
  • Children's Hospital of The King's Daughters
  • Children's Hopsital of Wisconsin, Corporate Center Suite 540
  • UZ Gent
  • UZ Leuven
  • University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases
  • Alberta Children's Hospital
  • Children's Hospital of Eastern Ontario
  • Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno
  • Fakultni nemocnice v Motole
  • Hotel Dieu- CHU Nantes
  • Hôpital Armand Trousseau
  • Universitatsklinikum Freiberg
  • Universitatsklinikum Essen
  • Schneider Children's Medical Center of Israel
  • UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
  • Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
  • Fondazione Policlinico Universitario A Gemelli
  • Samodzielny Publiczny Centralny Szpital Kliniczny
  • Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
  • Hospital de La Santa Creu i Sant Pau
  • Hospital Sant Joan de Deu
  • Hospital Universitari i Politecnic La Fe de Valencia
  • Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
  • Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust
  • Alder Hey Children's NHS Foundation Trust
  • Great Ormond Street Hospital (GOSH)
  • Royal Victoria Infirmary

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Casimersen

Golodirsen

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Number of Patients With Serious Adverse Events (SAEs)

Secondary Outcome Measures

Full Information

First Posted
May 10, 2018
Last Updated
August 16, 2023
Sponsor
Sarepta Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03532542
Brief Title
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

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Terminated
Why Stopped
Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There were no safety concerns with this study.
Study Start Date
August 2, 2018 (Actual)
Primary Completion Date
July 26, 2023 (Actual)
Study Completion Date
July 26, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
171 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Casimersen
Arm Type
Experimental
Arm Description
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.
Arm Title
Golodirsen
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Casimersen
Other Intervention Name(s)
SRP-4045, AMONDYS 45
Intervention Description
Casimersen solution for IV infusion
Intervention Type
Drug
Intervention Name(s)
Golodirsen
Other Intervention Name(s)
SRP-4053, VYONDYS 53
Intervention Description
Golodirsen solution for IV infusion
Primary Outcome Measure Information:
Title
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)

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
23 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Neuromuscular Research Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85028
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of California Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Stanford Neuroscience Health Center
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Rady Children's Hospital- San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
UF Health: University of Florida Clinical Research Center
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
NW Florida Clinical Research Group, LLC
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Ann and Robert H Lurie Childrens Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Iowa Childrens Hospital
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
St. Louis Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Las Vegas Clinic
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89145
Country
United States
Facility Name
University of Rochester Medical Center - Department of Neurology
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Abigail Wexner Research Institute at Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Shriners Hospital for Children
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Children's Medical Center Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah - PPDS
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Children's Hospital of The King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23510
Country
United States
Facility Name
Children's Hopsital of Wisconsin, Corporate Center Suite 540
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
UZ Gent
City
Gent
State/Province
Oost-Vlaanderen
ZIP/Postal Code
9000
Country
Belgium
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B6A8
Country
Canada
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno
City
Brno
ZIP/Postal Code
613 00
Country
Czechia
Facility Name
Fakultni nemocnice v Motole
City
Praha 5
ZIP/Postal Code
150 06
Country
Czechia
Facility Name
Hotel Dieu- CHU Nantes
City
Nantes Cedex
ZIP/Postal Code
44093
Country
France
Facility Name
Hôpital Armand Trousseau
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Universitatsklinikum Freiberg
City
Freiburg
State/Province
Baden-Wurttemberg
ZIP/Postal Code
70106
Country
Germany
Facility Name
Universitatsklinikum Essen
City
Essen
State/Province
Nordrhein-Westfalen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Schneider Children's Medical Center of Israel
City
Petach Tikva
ZIP/Postal Code
4920235
Country
Israel
Facility Name
UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
City
Genova
State/Province
Liguria
ZIP/Postal Code
16147
Country
Italy
Facility Name
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
City
Cona
ZIP/Postal Code
44124
Country
Italy
Facility Name
Fondazione Policlinico Universitario A Gemelli
City
Milano
ZIP/Postal Code
20123
Country
Italy
Facility Name
Samodzielny Publiczny Centralny Szpital Kliniczny
City
Warsaw
State/Province
Mazowieckie
ZIP/Postal Code
02-097
Country
Poland
Facility Name
Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Hospital de La Santa Creu i Sant Pau
City
Barcelona
State/Province
Cataluña
ZIP/Postal Code
08041
Country
Spain
Facility Name
Hospital Sant Joan de Deu
City
Barcelona
ZIP/Postal Code
8950
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
City
Goteborg
ZIP/Postal Code
41685
Country
Sweden
Facility Name
Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Facility Name
Alder Hey Children's NHS Foundation Trust
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
Great Ormond Street Hospital (GOSH)
City
London
ZIP/Postal Code
WC1N 1EH
Country
United Kingdom
Facility Name
Royal Victoria Infirmary
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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