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Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
SRP-4045
SRP-4053
Placebo
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, Ambulatory, Pediatric, Duchenne

Eligibility Criteria

6 Years - 13 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping
  • Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
  • Intact right and left biceps or 2 alternative upper muscle groups
  • Mean 6MWT ≥300 meters and ≤450 meters
  • Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted

Exclusion Criteria:

  • Treatment with gene therapy at any time
  • Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1
  • Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1
  • Major surgery within 3 months prior to Week 1
  • Presence of other clinically significant illness

Other inclusion/exclusion criteria may apply.

Sites / Locations

  • Neuromuscular Research Center
  • Children's Hospital Los Angeles
  • David Geffen School of Medicine, UCLA
  • Rady Children's Hospital San Diego/ UCSD
  • Stanford University School of Medicine/Medical Center
  • University of Florida
  • NW Florida Clinical Research Group, LLC
  • Center for Integrative Rare Disease Research (CIRDR)
  • Ann and Robert H. Lurie Children's Hospital of Chicago
  • University of Iowa Children's Hospital
  • University of Kansas, Medical Center
  • Boston Children's Hospital
  • St. Louis Children's Hospital
  • Las Vegas Clinic
  • University of Rochester Clinical Research Center
  • Cincinnati Children's Hospital Medical Center (CCHMC)
  • Nationwide Children's Hospital
  • Shriners Hospital for Children
  • Children's Hospital of Pittsburgh of UPMC
  • Children's Medical Center Dallas
  • University of Utah
  • Children's Hospital of the King's Daughters
  • Children's Hospital of Wisconsin
  • DOM Centro de Reumatologia
  • Royal Children's Hospital Melbourne
  • Queensland Children's Hospital
  • Children's Hospital at Westmead
  • Universitair Ziekenhuis Gent
  • Universitair Ziekenhuis Leuven
  • CHR de la Citadelle
  • University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
  • Alberta Childrens Hospital
  • Children's and Women's Health Centre of British Columbia
  • London Health Sciences Centre
  • Children's Hospital of Eastern Ontario
  • University Hospital Brno
  • Fakultni nemocnice v Motole
  • Rigshospitalet Copenhagen University Hospital
  • Hôpital Des Enfants
  • Reference Centre for Neuromuscular Diseases
  • Hôpital Armand Trousseau
  • Charité - Universitätsmedizin Berlin
  • Universitätsklinikum Essen
  • University Hospital Freiburg
  • IASO Children's Hospital
  • Ippokratio General Hospital of Thessaloniki
  • Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
  • Royal Instituite of Child Neurosciences
  • Deenanth Mangeshkar Hospital
  • The Children's University Hospital
  • Schneider Children's Medical Center of Israel
  • Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
  • Istituto Giannina Gaslini
  • Az Ospedaliera Universitaria Policlinico G Martino
  • Fondazione IRCCS Istituto Neurologico Carlo Besta
  • Policlinico Universitario A Gemelli
  • Seoul National University Hospital
  • Pusan National University Yangsan Hospital
  • Neurociencias Estudios Clínicos S.C
  • Instituto de Investigaciones Clínicas para la Salud A.C
  • Samodzielny Publiczny Centralny Szpital Kliniczny
  • Uniwersyteckie Centrum Kliniczne
  • Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation
  • State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
  • Clinic for Neurology and Psychiatry for Children and Youth
  • Hospital de La Santa Creu i Sant Pau
  • Hospital Sant Joan de Deu
  • Hospital Universitari i Politecnic La Fe de Valencia
  • Drottning Silvias Barn Och Ungdomssjukhus
  • Royal Hospital for Children (Glasgow)
  • Leeds Teaching Hospitals NHS Trust
  • Alder Hey Childrens Hospital
  • Great Ormond Street Hospital (GOSH)
  • Royal Victoria Infirmary
  • John Radcliffe Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

SRP-4045

SRP-4053

Placebo followed by SRP-4045 or SRP-4053

Arm Description

Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Outcomes

Primary Outcome Measures

Change From Baseline in the Total Distance Walked During 6MWT at Week 96

Secondary Outcome Measures

Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period)
Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
Participant's Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore
The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, the participant's ability to rise independently from the floor (without external support) will be reported as an NSAA subscore of "2" (without modification) or "1" (Gower's maneuver).
Time to Loss of Ambulation (LOA)
Change From Baseline in the NSAA Total Score at Week 96 and Week 144
The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, participants will be asked to perform 17 different functional activities, including a 10 meter walk/run, rising from a sit to standing, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participants will be graded as follows: 2 = achieves goal without any assistance; 1 = modified method but achieves goal independent of physical assistance from another person; and 0 = unable to achieve goal independently. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144

Full Information

First Posted
July 14, 2015
Last Updated
February 16, 2023
Sponsor
Sarepta Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02500381
Brief Title
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
Acronym
ESSENCE
Official Title
A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 28, 2016 (Actual)
Primary Completion Date
October 3, 2025 (Anticipated)
Study Completion Date
October 3, 2025 (Anticipated)

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 efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
Detailed Description
This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible participants with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 milligrams/kilograms (mg/kg) SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group) or placebo for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open-label extension period in which all participants will receive open-label active treatment for 48 weeks (up to Week 144 of study). The study will enroll approximately 222 participants. Twice as many participants will be randomized to receive active treatment as will receive placebo (2:1 randomization). Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests, such as the 6-minute walk test (6MWT). All participants will undergo a muscle biopsy at baseline and a second muscle biopsy either at Week 48 or Week 96. Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study. Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of both drugs.

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, Ambulatory, Pediatric, Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Part 1 is double-blind and randomized; Part 2 is open-label.
Allocation
Randomized
Enrollment
229 (Actual)

8. Arms, Groups, and Interventions

Arm Title
SRP-4045
Arm Type
Experimental
Arm Description
Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
Arm Title
SRP-4053
Arm Type
Experimental
Arm Description
Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
Arm Title
Placebo followed by SRP-4045 or SRP-4053
Arm Type
Placebo Comparator
Arm Description
Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
Intervention Type
Drug
Intervention Name(s)
SRP-4045
Other Intervention Name(s)
Casimersen, AMONDYS 45
Intervention Description
SRP-4045 solution for IV infusion
Intervention Type
Drug
Intervention Name(s)
SRP-4053
Other Intervention Name(s)
Golodirsen, VYONDYS 53
Intervention Description
SRP-4053 solution for IV infusion
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
SRP-4045 or SRP-4053 placebo-matching solution for IV infusion
Primary Outcome Measure Information:
Title
Change From Baseline in the Total Distance Walked During 6MWT at Week 96
Time Frame
Baseline, Week 96
Secondary Outcome Measure Information:
Title
Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period)
Time Frame
Baseline, Week 144
Title
Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
Time Frame
Baseline, Week 48 or Week 96
Title
Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
Time Frame
Baseline, Week 48 or Week 96
Title
Participant's Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore
Description
The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, the participant's ability to rise independently from the floor (without external support) will be reported as an NSAA subscore of "2" (without modification) or "1" (Gower's maneuver).
Time Frame
Week 96, Week 144
Title
Time to Loss of Ambulation (LOA)
Time Frame
Baseline, Week 96, and Week 144
Title
Change From Baseline in the NSAA Total Score at Week 96 and Week 144
Description
The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, participants will be asked to perform 17 different functional activities, including a 10 meter walk/run, rising from a sit to standing, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participants will be graded as follows: 2 = achieves goal without any assistance; 1 = modified method but achieves goal independent of physical assistance from another person; and 0 = unable to achieve goal independently. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Time Frame
Baseline, Week 96 and Week 144
Title
Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144
Time Frame
Baseline, Week 96 and Week 144

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). Intact right and left biceps or 2 alternative upper muscle groups Mean 6MWT ≥300 meters and ≤450 meters Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted Exclusion Criteria: Treatment with gene therapy at any time Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1 Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1 Major surgery within 3 months prior to Week 1 Presence of other clinically significant illness Other inclusion/exclusion criteria may 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
David Geffen School of Medicine, UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Rady Children's Hospital San Diego/ UCSD
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Stanford University School of Medicine/Medical Center
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
University of Florida
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
Center for Integrative Rare Disease Research (CIRDR)
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Iowa Children's 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
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 Clinical Research Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center (CCHMC)
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
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
75235
Country
United States
Facility Name
University of Utah
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
23507
Country
United States
Facility Name
Children's Hospital of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
DOM Centro de Reumatologia
City
Ciudad Autonoma de Buenos Aires
ZIP/Postal Code
1111
Country
Argentina
Facility Name
Royal Children's Hospital Melbourne
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
ZIP/Postal Code
4101
Country
Australia
Facility Name
Children's Hospital at Westmead
City
Westmead
ZIP/Postal Code
2145
Country
Australia
Facility Name
Universitair Ziekenhuis Gent
City
Ghent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Universitair Ziekenhuis Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
CHR de la Citadelle
City
Liège
ZIP/Postal Code
4000
Country
Belgium
Facility Name
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
City
Sofia
State/Province
Sofia-Grad
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
Alberta Childrens Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Facility Name
Children's and Women's Health Centre of British Columbia
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
University Hospital Brno
City
Brno
ZIP/Postal Code
61300
Country
Czechia
Facility Name
Fakultni nemocnice v Motole
City
Praha
ZIP/Postal Code
15008
Country
Czechia
Facility Name
Rigshospitalet Copenhagen University Hospital
City
København Ø
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Hôpital Des Enfants
City
Toulouse
State/Province
Haute-Garonne
ZIP/Postal Code
31059
Country
France
Facility Name
Reference Centre for Neuromuscular Diseases
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Hôpital Armand Trousseau
City
Paris
ZIP/Postal Code
75012
Country
France
Facility Name
Charité - Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Universitätsklinikum Essen
City
Essen
ZIP/Postal Code
45122
Country
Germany
Facility Name
University Hospital Freiburg
City
Freiburg
ZIP/Postal Code
79106
Country
Germany
Facility Name
IASO Children's Hospital
City
Maroussi
ZIP/Postal Code
15123
Country
Greece
Facility Name
Ippokratio General Hospital of Thessaloniki
City
Thessaloniki
ZIP/Postal Code
54642
Country
Greece
Facility Name
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Royal Instituite of Child Neurosciences
City
Ahmedabad
State/Province
Gujarat
ZIP/Postal Code
380054
Country
India
Facility Name
Deenanth Mangeshkar Hospital
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411004
Country
India
Facility Name
The Children's University Hospital
City
Dublin
ZIP/Postal Code
D1
Country
Ireland
Facility Name
Schneider Children's Medical Center of Israel
City
Petah Tikvah
ZIP/Postal Code
49102
Country
Israel
Facility Name
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
City
Ferrara
ZIP/Postal Code
44124
Country
Italy
Facility Name
Istituto Giannina Gaslini
City
Genoa
ZIP/Postal Code
16147
Country
Italy
Facility Name
Az Ospedaliera Universitaria Policlinico G Martino
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
Fondazione IRCCS Istituto Neurologico Carlo Besta
City
Milano
ZIP/Postal Code
20133
Country
Italy
Facility Name
Policlinico Universitario A Gemelli
City
Rome
ZIP/Postal Code
00168
Country
Italy
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Pusan National University Yangsan Hospital
City
Yangsan
ZIP/Postal Code
50612
Country
Korea, Republic of
Facility Name
Neurociencias Estudios Clínicos S.C
City
Culiacán
ZIP/Postal Code
80020
Country
Mexico
Facility Name
Instituto de Investigaciones Clínicas para la Salud A.C
City
Durango
ZIP/Postal Code
34000
Country
Mexico
Facility Name
Samodzielny Publiczny Centralny Szpital Kliniczny
City
Warsaw
State/Province
Mazowieckie
ZIP/Postal Code
02-097
Country
Poland
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation
City
Moscow
ZIP/Postal Code
125412
Country
Russian Federation
Facility Name
State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
City
Yekaterinburg
Country
Russian Federation
Facility Name
Clinic for Neurology and Psychiatry for Children and Youth
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Facility Name
Hospital de La Santa Creu i Sant Pau
City
Barcelona
ZIP/Postal Code
08041
Country
Spain
Facility Name
Hospital Sant Joan de Deu
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
Country
Spain
Facility Name
Drottning Silvias Barn Och Ungdomssjukhus
City
Göteborg
ZIP/Postal Code
SE-41685
Country
Sweden
Facility Name
Royal Hospital for Children (Glasgow)
City
Glasgow
ZIP/Postal Code
G51 4TF
Country
United Kingdom
Facility Name
Leeds Teaching Hospitals NHS Trust
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Facility Name
Alder Hey Childrens Hospital
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
Facility Name
John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Undecided
Citations:
PubMed Identifier
34105177
Citation
Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.
Results Reference
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Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

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