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A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Primary Purpose

Muscular Dystrophy, Duchenne

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

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne muscular dystrophy, Exon Skipping, Exon 51, North Star Ambulatory Assessment, Ambulatory, DMD, Pediatric, Duchenne, EXONDYS, MIS51ON

Eligibility Criteria

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

Inclusion Criteria:

  • Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
  • Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.
  • Able to walk independently without assistive devices.
  • Have intact right and left biceps muscles or an alternative upper arm muscle group.
  • Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study.
  • For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening.

Exclusion Criteria:

  • Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
  • Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; and Eteplirsen (previous or current use).
  • Major surgery within 3 months prior to randomization.
  • Presence of any other significant neuromuscular or genetic disease other than DMD.
  • Presence of any known impairment of renal function and/or other clinically significant illness.
  • Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than <50% on the screening echocardiogram or Fridericia's correction formula (QTcF) ≥450 millisecond based on the screening electrocardiograms (ECGs).

Other inclusion/exclusion criteria apply.

Sites / Locations

  • University of Alabama at BirminghamRecruiting
  • University of FloridaRecruiting
  • Rare Disease Research, LLCRecruiting
  • Children's Hospital - London Health Sciences Centre
  • Children's Hospital of Eastern Ontario
  • Hospital Universitario San IgnacioRecruiting
  • Instituto Neurologico de Colombia (INDEC)Recruiting
  • Hospital Pablo Tobón UribeRecruiting
  • Brno Klinika detske neurologieRecruiting
  • Fakultni nemocnice v MotoleRecruiting
  • Rigshospitalet Copenhagen University HospitalRecruiting
  • Hopital Femme Mere EnfantRecruiting
  • Hopital Armand TrousseauRecruiting
  • CHRU de StrasbourgRecruiting
  • Charité Universitätsmedizin Berlin CVKRecruiting
  • Universitätsklinikum EssenRecruiting
  • Universitätsklinikum FreiburgRecruiting
  • IASO Children's HospitalRecruiting
  • Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek IntezeteRecruiting
  • Royal Institute of Child NeurosciencesRecruiting
  • Aster RV HospitalRecruiting
  • Nizam's Institute of Medical SciencesRecruiting
  • Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)Recruiting
  • All India Institute of Medical SciencesRecruiting
  • Sir Ganga Ram HospitalRecruiting
  • Deenanath Mangeshkar Hospital & Research CentreRecruiting
  • Christian Medical CollegeRecruiting
  • Children's Health Ireland (CHI) at Temple StreetRecruiting
  • IRCCS Instituto Gianna GasliniRecruiting
  • The Specialty Hospital (TSH)/Advanced Clinical CenterRecruiting
  • Istiklal Hosptial (IST)Recruiting
  • Irbid Specialty HospitalRecruiting
  • Pharmaceutical Research Center/Jordan University of Science and TechnologyRecruiting
  • Kyungpook National University Chilgok HospitalRecruiting
  • Seoul National University HospitalRecruiting
  • Samsung Medical CenterRecruiting
  • Pusan National University Yangsan HospitalRecruiting
  • Neurociencias Estudios Clínicos S.C.Recruiting
  • Instituto de Investigaciones Clinicas para la Salud A.CRecruiting
  • Leids Universitair Medisch CentrumRecruiting
  • Radboud University Nijmegen Medical CentreRecruiting
  • New Zealand Clinical Research - AucklandRecruiting
  • Oslo Universitetssykehus HF RikshospitaletRecruiting
  • Children's Department and Department for Children's Habilitation at Stavanger University HospitalRecruiting
  • Klinika Neurologii RozwojowejRecruiting
  • National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"Recruiting
  • Clinic for Neurology and Psychiatry for Children and YouthRecruiting
  • University Children's HospitalRecruiting
  • Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"Recruiting
  • University Medical Centre LjubljanaRecruiting
  • Hospital Sant Joan de DeuRecruiting
  • Hospital Universitari i Politecnic La Fe de ValenciaRecruiting
  • Universitätsspital BaselRecruiting
  • Kaohsiung Medical UniversityRecruiting
  • National Taiwan University HospitalRecruiting
  • Akdeniz Universitesi Tip FakultesiRecruiting
  • Mersin University Medical FacultyRecruiting
  • Leeds Teaching Hospitals NHS TrustRecruiting
  • Birmingham Heartlands HospitalRecruiting
  • UCL Institute of Child Health Great Ormond StreetRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Active Comparator

Experimental

Experimental

Arm Label

Part 1: Eteplirsen

Part 2: Eteplirsen 30 mg/kg

Part 2: Eteplirsen 100 mg/kg

Part 2: Eteplirsen 200 mg/kg

Arm Description

Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks.

Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.

Randomized participants will receive eteplirsen 100 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.

Randomized participants will receive eteplirsen 200 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.

Outcomes

Primary Outcome Measures

Part 1: Incidence of Adverse Events (AEs)
Part 2: Change From Baseline in the NSAA Total Score at Week 144

Secondary Outcome Measures

Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test
Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
Part 2: Change from Baseline in Forced Vital Capacity Percent Predicted (FVC%p) at Week 144
Part 2: Time to Loss of Ambulation (LOA)
Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression
Part 2: Incidence of Adverse Events (AEs)
Part 2: Pharmacokinetic (PK) Plasma Concentration of Eteplirsen

Full Information

First Posted
June 18, 2019
Last Updated
October 4, 2023
Sponsor
Sarepta Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03992430
Brief Title
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Official Title
A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 13, 2020 (Actual)
Primary Completion Date
November 30, 2024 (Anticipated)
Study Completion Date
November 30, 2024 (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
This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne
Keywords
Duchenne muscular dystrophy, Exon Skipping, Exon 51, North Star Ambulatory Assessment, Ambulatory, DMD, Pediatric, Duchenne, EXONDYS, MIS51ON

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Part 1 is open-label, dose escalation; Part 2 is double-blind, dose finding, and dose comparison
Allocation
Randomized
Enrollment
154 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Part 1: Eteplirsen
Arm Type
Experimental
Arm Description
Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks.
Arm Title
Part 2: Eteplirsen 30 mg/kg
Arm Type
Active Comparator
Arm Description
Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.
Arm Title
Part 2: Eteplirsen 100 mg/kg
Arm Type
Experimental
Arm Description
Randomized participants will receive eteplirsen 100 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.
Arm Title
Part 2: Eteplirsen 200 mg/kg
Arm Type
Experimental
Arm Description
Randomized participants will receive eteplirsen 200 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.
Intervention Type
Drug
Intervention Name(s)
Eteplirsen
Other Intervention Name(s)
AVI-4658, EXONDYS 51, EXONDYS
Intervention Description
Solution for intravenous (IV) infusion.
Primary Outcome Measure Information:
Title
Part 1: Incidence of Adverse Events (AEs)
Time Frame
Up to Week 148
Title
Part 2: Change From Baseline in the NSAA Total Score at Week 144
Time Frame
Baseline, Week 144
Secondary Outcome Measure Information:
Title
Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test
Time Frame
Baseline, Week 144
Title
Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
Time Frame
Baseline, Week 144
Title
Part 2: Change from Baseline in Forced Vital Capacity Percent Predicted (FVC%p) at Week 144
Time Frame
Baseline, Week 144
Title
Part 2: Time to Loss of Ambulation (LOA)
Time Frame
Baseline up to Week 144
Title
Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression
Time Frame
Baseline, Postdose (at Week 24, Week 48, or Week 144)
Title
Part 2: Incidence of Adverse Events (AEs)
Time Frame
Baseline up to Week 148
Title
Part 2: Pharmacokinetic (PK) Plasma Concentration of Eteplirsen
Time Frame
0 (predose) to 2 hours postdose up to Week 144

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping. Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit. Able to walk independently without assistive devices. Have intact right and left biceps muscles or an alternative upper arm muscle group. Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study. For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening. Exclusion Criteria: Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization. Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; and Eteplirsen (previous or current use). Major surgery within 3 months prior to randomization. Presence of any other significant neuromuscular or genetic disease other than DMD. Presence of any known impairment of renal function and/or other clinically significant illness. Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than <50% on the screening echocardiogram or Fridericia's correction formula (QTcF) ≥450 millisecond based on the screening electrocardiograms (ECGs). Other inclusion/exclusion criteria apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Phone
1-888-SAREPTA (1-888-727-3782)
Email
SareptAlly@sarepta.com
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
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Phone
205-638-2551
Email
malopez@uabmc.edu
First Name & Middle Initial & Last Name & Degree
Michael Lopez
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610-3010
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Phone
352-294-5757
Email
barry.byrne@ufl.edu
First Name & Middle Initial & Last Name & Degree
Barry Byrne
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Phone
678-883-6897
Email
Info@rarediseaseresearch.com
First Name & Middle Initial & Last Name & Degree
Han Phan, MD
Facility Name
Children's Hospital - London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Individual Site Status
Withdrawn
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Individual Site Status
Withdrawn
Facility Name
Hospital Universitario San Ignacio
City
Bogotá
ZIP/Postal Code
110231
Country
Colombia
Individual Site Status
Recruiting
Facility Contact:
Email
SareptAlly@sarepta.com
First Name & Middle Initial & Last Name & Degree
Fernando Suarez-Obando
Facility Name
Instituto Neurologico de Colombia (INDEC)
City
Medellin
ZIP/Postal Code
50012
Country
Colombia
Individual Site Status
Recruiting
Facility Contact:
Phone
3008280830
Email
carlos.franco@neurologico.org.co
First Name & Middle Initial & Last Name & Degree
Carlos Alberto Franco Restrepo
Facility Name
Hospital Pablo Tobón Uribe
City
Medellin
ZIP/Postal Code
50034
Country
Colombia
Individual Site Status
Recruiting
Facility Contact:
Phone
+57 3146470298
Email
smesar@hptu.org.co
First Name & Middle Initial & Last Name & Degree
Sandra Catalina Mesa Restrepo
Facility Name
Brno Klinika detske neurologie
City
Brno
ZIP/Postal Code
613 00
Country
Czechia
Individual Site Status
Recruiting
Facility Contact:
Phone
420532 234 581; 420737 778 483
Email
jurikova.lenka@fnbrno.cz; lenkamrazo@seznam.cz
First Name & Middle Initial & Last Name & Degree
Lenka Jurikova, MD
Facility Name
Fakultni nemocnice v Motole
City
Praha 5
ZIP/Postal Code
150 06
Country
Czechia
Individual Site Status
Recruiting
Facility Contact:
Phone
+420224433301
Email
jana.haberlova@fnmotol.cz
First Name & Middle Initial & Last Name & Degree
Jana Haberlov, MD
Facility Name
Rigshospitalet Copenhagen University Hospital
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
Phone
+4535454467
Email
malene.landbo.02.boerresen@regionh.dk
First Name & Middle Initial & Last Name & Degree
Malene Landbo Borresen
Facility Name
Hopital Femme Mere Enfant
City
Bron
ZIP/Postal Code
69677
Country
France
Individual Site Status
Recruiting
Facility Contact:
Phone
+33472681273
Email
aurelie.portefaix@chu-lyon.fr
First Name & Middle Initial & Last Name & Degree
Aurélie Portefaix
Facility Name
Hopital Armand Trousseau
City
Paris
ZIP/Postal Code
75571
Country
France
Individual Site Status
Recruiting
Facility Contact:
Phone
+33 (0)1 71 73 80 50
Email
a.seferian@institut-myologie.org
First Name & Middle Initial & Last Name & Degree
Andreea Seferian, MD
Facility Name
CHRU de Strasbourg
City
Strasbourg
ZIP/Postal Code
67098
Country
France
Individual Site Status
Recruiting
Facility Contact:
Phone
+33 3 88 12 77 43
Email
vincent.laugel@chru-strasbourg.fr
First Name & Middle Initial & Last Name & Degree
Vincent Laugel
Facility Name
Charité Universitätsmedizin Berlin CVK
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
Phone
+49 30 450 666 279
Email
Claudia.Weiss@charite.de
First Name & Middle Initial & Last Name & Degree
Claudia Weiss
Facility Name
Universitätsklinikum Essen
City
Essen
ZIP/Postal Code
45147
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
Email
spz-studienteam@uk-essen.de
First Name & Middle Initial & Last Name & Degree
Andrea Gangfuss
Facility Name
Universitätsklinikum Freiburg
City
Freiburg
ZIP/Postal Code
79106
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Janbernd Kirschner
Facility Name
IASO Children's Hospital
City
Marousi
State/Province
Attiki
ZIP/Postal Code
151 23
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
Phone
0030 - 6932246726
Email
theon@otenet.gr
First Name & Middle Initial & Last Name & Degree
Antigone S. Papavailiou, MD, PhD, FAAP
Facility Name
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
City
Budapest
ZIP/Postal Code
1082
Country
Hungary
Individual Site Status
Recruiting
Facility Contact:
Phone
+36 20 6632513
Email
molnarmj@gmail.com
First Name & Middle Initial & Last Name & Degree
Maria Molnar
Facility Name
Royal Institute of Child Neurosciences
City
Ahmedabad
ZIP/Postal Code
380054
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+919909960555
Email
sidh909@hotmail.com
First Name & Middle Initial & Last Name & Degree
Siddarth Shah
Facility Name
Aster RV Hospital
City
Bengaluru
ZIP/Postal Code
560078
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+91-9008360858
Email
annagnesmathew@yahoo.co.in
First Name & Middle Initial & Last Name & Degree
Ann Mathew
Facility Name
Nizam's Institute of Medical Sciences
City
Hyderabad
ZIP/Postal Code
500082
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+919246589899
Email
surmukh99@gmail.com
First Name & Middle Initial & Last Name & Degree
Surya Prabha Turaga
Facility Name
Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)
City
Madurai
ZIP/Postal Code
626022
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+91-9840301991
Email
Raghavank27@gmail.com
First Name & Middle Initial & Last Name & Degree
Raghavan Kadalraja
Facility Name
All India Institute of Medical Sciences
City
New Delhi
ZIP/Postal Code
110029
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+91-9810386847
Email
sheffaligulati@gmail.com
First Name & Middle Initial & Last Name & Degree
Sheffali Gulati, MD
Facility Name
Sir Ganga Ram Hospital
City
New Delhi
ZIP/Postal Code
110060
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+919811869192
Email
ratnadpuri@yahoo.com
First Name & Middle Initial & Last Name & Degree
Ratna Puri, MD
Facility Name
Deenanath Mangeshkar Hospital & Research Centre
City
Pune
ZIP/Postal Code
411004
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+919324316759
Email
umeshkalane@yahoo.com
First Name & Middle Initial & Last Name & Degree
Umesh Kalane
Facility Name
Christian Medical College
City
Vellore
ZIP/Postal Code
632004
Country
India
Individual Site Status
Recruiting
Facility Contact:
Phone
+919442611622
Email
doc_ys@yahoo.co.in
First Name & Middle Initial & Last Name & Degree
Sangeetha Yoganathan, MD
Facility Name
Children's Health Ireland (CHI) at Temple Street
City
Dublin 1
ZIP/Postal Code
D01 XD99
Country
Ireland
Individual Site Status
Recruiting
Facility Contact:
Phone
+35318784722
Email
declan.orourke@cuh.ie
First Name & Middle Initial & Last Name & Degree
Declan O'Rourke, MD
Facility Name
IRCCS Instituto Gianna Gaslini
City
Genova
ZIP/Postal Code
16147
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
Phone
+39 0105636260
Email
claudiobruno@gmail.com
First Name & Middle Initial & Last Name & Degree
Claudio Bruno
Facility Name
The Specialty Hospital (TSH)/Advanced Clinical Center
City
Amman
ZIP/Postal Code
11194
Country
Jordan
Individual Site Status
Recruiting
Facility Contact:
Phone
+962-7 9034 2070
Email
dr.mai.bader@hotmail.com
First Name & Middle Initial & Last Name & Degree
May Bader
Facility Name
Istiklal Hosptial (IST)
City
Amman
ZIP/Postal Code
11196
Country
Jordan
Individual Site Status
Recruiting
Facility Contact:
Phone
07 9649 9696
Email
abdee4400@yahoo.com
First Name & Middle Initial & Last Name & Degree
Abdee Ryalat
Facility Name
Irbid Specialty Hospital
City
Irbid
ZIP/Postal Code
22110
Country
Jordan
Individual Site Status
Recruiting
Facility Contact:
Phone
+962799050392
Email
Shurman_Abdalla@yahoo.com
First Name & Middle Initial & Last Name & Degree
Abdalla Al Shurman
Facility Name
Pharmaceutical Research Center/Jordan University of Science and Technology
City
Irbid
ZIP/Postal Code
22110
Country
Jordan
Individual Site Status
Recruiting
Facility Contact:
Phone
+962 790247496
Email
maalmomani3@just.edu.jo
First Name & Middle Initial & Last Name & Degree
Miral AlMomani
Facility Name
Kyungpook National University Chilgok Hospital
City
Daegu
ZIP/Postal Code
41404
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
Phone
+ 82532003278
Email
jinforeva@gmail.com
First Name & Middle Initial & Last Name & Degree
Prof. Jin-Sung Park, MD
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
Email
SareptAlly@sarepta.com
First Name & Middle Initial & Last Name & Degree
Hee Jong Chae, MD
Facility Name
Samsung Medical Center
City
Seoul
ZIP/Postal Code
6351
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
JeeHun Lee, MD
Phone
(+) 82 2-3410-3539
Email
jhnr.lee@samsung.com
First Name & Middle Initial & Last Name & Degree
Jee Hun Lee, MD
Facility Name
Pusan National University Yangsan Hospital
City
Yangsan
ZIP/Postal Code
50612
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
Phone
+82553602122
Email
shinzh@pusan.ac.kr
First Name & Middle Initial & Last Name & Degree
Jin-Hong Shin, MD
Facility Name
Neurociencias Estudios Clínicos S.C.
City
Culiacán
State/Province
Sinaloa
ZIP/Postal Code
80020
Country
Mexico
Individual Site Status
Recruiting
Facility Contact:
Phone
+52 667-758-7952
Email
elmerlopez@hotmail.com
First Name & Middle Initial & Last Name & Degree
Elmer L Meza, MD
Facility Name
Instituto de Investigaciones Clinicas para la Salud A.C
City
Durango
ZIP/Postal Code
34000
Country
Mexico
Individual Site Status
Recruiting
Facility Contact:
Phone
+52 618-134-2987
Email
contacto@iics.com.mx
First Name & Middle Initial & Last Name & Degree
Marco M Gurrola, MD
Facility Name
Leids Universitair Medisch Centrum
City
Leiden
ZIP/Postal Code
2333 ZC
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
Phone
+31715262197
Email
E.H.Niks@lumc.nl
First Name & Middle Initial & Last Name & Degree
Erik Niks
Facility Name
Radboud University Nijmegen Medical Centre
City
Nijmegen
ZIP/Postal Code
6525GA
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
Email
saskia.houwen@radboudumc.nl
First Name & Middle Initial & Last Name & Degree
Saskia Houwen-van Opstal
Facility Name
New Zealand Clinical Research - Auckland
City
Auckland
ZIP/Postal Code
1010
Country
New Zealand
Individual Site Status
Recruiting
Facility Contact:
Phone
+6493074931
Email
paedneuro@adhb.govt.nz
First Name & Middle Initial & Last Name & Degree
Gina O'Grady, MD
Facility Name
Oslo Universitetssykehus HF Rikshospitalet
City
Oslo
ZIP/Postal Code
0450
Country
Norway
Individual Site Status
Recruiting
Facility Contact:
Phone
+47 91372036
Email
seawal@ous-hf.no
First Name & Middle Initial & Last Name & Degree
Sean Wallace, MD
Facility Name
Children's Department and Department for Children's Habilitation at Stavanger University Hospital
City
Stavanger
ZIP/Postal Code
4011
Country
Norway
Individual Site Status
Recruiting
Facility Contact:
Email
SareptAlly@sarepta.com
First Name & Middle Initial & Last Name & Degree
Katja Sara Holmefjord
Facility Name
Klinika Neurologii Rozwojowej
City
Gdansk
State/Province
Pomorskie
ZIP/Postal Code
80-211
Country
Poland
Individual Site Status
Recruiting
Facility Contact:
Phone
+48 58 349 23 96
Email
mmazur@gumed.edu.pl
First Name & Middle Initial & Last Name & Degree
Maria Mazurkiewicz-Beldzinska, MD
Facility Name
National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"
City
Bucharest
ZIP/Postal Code
41408
Country
Romania
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Madalina Leanca
Facility Name
Clinic for Neurology and Psychiatry for Children and Youth
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
Email
kosacana@gmail.com
First Name & Middle Initial & Last Name & Degree
Ana Kosac, MD
Facility Name
University Children's Hospital
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
Email
dimitrije.nikolic@gmail.com
First Name & Middle Initial & Last Name & Degree
Dimitrije Nikolic, MD
Facility Name
Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"
City
Belgrade
ZIP/Postal Code
190133
Country
Serbia
Individual Site Status
Recruiting
Facility Contact:
Phone
+381641608264
Email
gocavlahovic@yahoo.com
First Name & Middle Initial & Last Name & Degree
Gordana Kovacevic, MD
Facility Name
University Medical Centre Ljubljana
City
Ljubljana
ZIP/Postal Code
1000
Country
Slovenia
Individual Site Status
Recruiting
Facility Contact:
Phone
00-386-15229273
Email
damjan.osredkar@kclj.si
First Name & Middle Initial & Last Name & Degree
Damjan Osredkar
Facility Name
Hospital Sant Joan de Deu
City
Barcelona
ZIP/Postal Code
8950
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
Phone
+34 93-600-9733
Email
maria.valle@sid.es
First Name & Middle Initial & Last Name & Degree
Andres Nascimiento
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
Phone
0034 9-1244153
Email
muelas_nur@gra.es
First Name & Middle Initial & Last Name & Degree
Nuria Muelas, MD
Facility Name
Universitätsspital Basel
City
Basel
ZIP/Postal Code
4031
Country
Switzerland
Individual Site Status
Recruiting
Facility Contact:
Email
dirk.fischer@ukbb.ch
First Name & Middle Initial & Last Name & Degree
Dirk Fischer
Facility Name
Kaohsiung Medical University
City
Kaohsiung
ZIP/Postal Code
80756
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
Phone
+886975356756
Email
yjjong@gap.kmu.edu.tw
First Name & Middle Initial & Last Name & Degree
Yuh-Jyh Jong, MD
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10071
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
Email
majalis.tw@gmail.com
First Name & Middle Initial & Last Name & Degree
Wang-Tso Lee, MD, PhD
Facility Name
Akdeniz Universitesi Tip Fakultesi
City
Antalya
ZIP/Postal Code
07059
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
Phone
+90-553-5819641
Email
oduman@akdeniz.edu.tr
First Name & Middle Initial & Last Name & Degree
Ozgur Duman, MD
Facility Name
Mersin University Medical Faculty
City
Mersin
ZIP/Postal Code
33110
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
Phone
+90-505-2677499
Email
mustafakomur@mersin.edu.tr
First Name & Middle Initial & Last Name & Degree
Mustafa Komur
Facility Name
Leeds Teaching Hospitals NHS Trust
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
anne-marie.childs@nhs.net
First Name & Middle Initial & Last Name & Degree
Anne-Marie Childs, MD
Facility Name
Birmingham Heartlands Hospital
City
Birmingham
ZIP/Postal Code
B9 5SS
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
SareptAlly@sarepta.com
First Name & Middle Initial & Last Name & Degree
Zoya Alhaswani, MD
Facility Name
UCL Institute of Child Health Great Ormond Street
City
London
ZIP/Postal Code
WC1N 1EH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Phone
+442079052111
Email
f.muntoni@ucl.ac.uk
First Name & Middle Initial & Last Name & Degree
Francesco Muntoni, MD

12. IPD Sharing Statement

Links:
URL
https://clinicaltrials.sarepta.com/MIS51ON
Description
Click here to access the website, clinicaltrials.sarepta.com/MIS51ON, for additional information for the study.

Learn more about this trial

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

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