Study of Eteplirsen in DMD Patients



Status:Active, not recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:7 - 16
Updated:11/28/2018
Start Date:November 17, 2014
End Date:May 1, 2019

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An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658)
in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
Additional objectives include evaluation of safety, biomarkers and the long-term effects of
eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen
in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic
deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD
patients not amenable to exon 51 skipping (untreated group). Following primary efficacy
endpoints, dosing will continue to week 144 to evaluate the long term effects of eteplirsen.

Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg
Eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks). Patients in
the untreated group will not receive treatment.

Clinical efficacy will be assessed at regularly scheduled study visits, including functional
tests such as the six minute walk test. Patients in the treated group will undergo a muscle
biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the
untreated group will not undergo muscle biopsy.

Safety, including adverse event monitoring and routine laboratory assessments, will be
continuously monitored for all patients.

Inclusion Criteria:

- Male 7-16 years old

- Diagnosed with DMD, genotypically confirmed

- Stable dose of corticosteroids for at least 24 weeks

- Have intact right and left alternative upper muscle groups

- Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)

- Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and
LVEF of greater than 50%

Exclusion Criteria:

- Previous treatment with drisapersen or any other RNA antisense agent or any gene
therapy within the last 6 months

- Participation in any other DMD interventional clinical study within 12 weeks

- Major surgery within 3 months

- Presence of other clinically significant illness

- Major change in the physical therapy regime within 3 months

Other inclusion/exclusion criteria apply.
We found this trial at
37
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201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Principal Investigator: Han C Phan, MD
Phone: 404-393-3205
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Principal Investigator: Kathryn Wagner, MD
Phone: 443-923-7318
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Fawn Leigh, MD
Phone: 617-643-4662
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Jerry R Mendell, MD
Phone: 614-722-2558
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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282 Washington St
Hartford, Connecticut 06106
(860) 545-9000
Principal Investigator: Gyula Acsadi, MD
Phone: 860-837-5881
Connecticut Children's Medical Center Connecticut Children’s Medical Center is a nationally recognized, 187-bed not-for-profit children’s...
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Gerald Raymond, MD
Phone: 612-625-4882
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Principal Investigator: William Burnette, MD
Phone: 615-322-8957
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Gihan Tennekoon, MD
Phone: 267-425-2111
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Hoda Z Abdel-Hamid, MD
Phone: 412-692-5176
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Russell Butterfield, MD
Phone: 801-585-9399
University of Utah Research is a major component in the life of the U benefiting...
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Atlanta, Georgia 30318
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Julie Parsons, MD
Phone: 720-777-3293
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Boston, Massachusetts 02115
Principal Investigator: Basil Darras, MD
Phone: 857-218-4677
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Charlotte, North Carolina 28203
Principal Investigator: Ibrahim Binalsheikh, MD
Phone: 704-446-3491
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Nancy Kuntz, MD
Phone: 312-227-3813
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Cincinnati, Ohio 45229
Principal Investigator: Cuixia Tian, MD
Phone: 513-636-5517
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1801 Inwood Rd
Dallas, Texas 75390
(214) 645-3300
Principal Investigator: Susan T Iannaccone, MD
Phone: 214-456-2463
University of Texas Southwestern Medical Center UT Southwestern is an academic medical center, world-renowned for...
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3901 Beaubien St
Detroit, Michigan 48201
(313) 745-5437
Principal Investigator: Huiyuan Jiang, MD
Phone: 313-966-0488
Children's Hospital of Michigan Since 1886, the Children's Hospital of Michigan has been dedicated to...
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Gainesville, Florida 32610
Principal Investigator: Barry J Byrne, MD
Phone: 352-273-8218
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Gulf Breeze, Florida 32561
Principal Investigator: Ben Renfroe, MD
Phone: 850-221-5116
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Hershey, Pennsylvania 17033
Principal Investigator: Matthew Wicklund, MD
Phone: 717-531-0003
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6621 Fannin St
Houston, Texas 77030
(832) 824-1000
Principal Investigator: Tim Lotze, MD
Phone: 832-822-1268
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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Iowa City, Iowa 52242
Principal Investigator: Katherine Mathews, MD
Phone: 319-384-9619
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Richard Barohn, MD
Phone: 913-945-9922
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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1 Medical Center Dr
Lebanon, New Hampshire 03756
 (603) 650-5000
Principal Investigator: Linda A Specht, MD
Phone: 603-653-9948
Dartmouth Hitchcock Medical Center Dartmouth-Hitchcock is a national leader in patient-centered health care and building...
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10833 Le Conte Ave
Los Angeles, California 90095
(310) 825-4321
Principal Investigator: Perry Shieh, MD
Phone: 310-825-3264
David Geffen School of Medicine, UCLA In 2002 Mr. David Geffen announced a $200 million...
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Principal Investigator: Richard Finkel, MD
Phone: 407-650-7523
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Phoenix, Arizona 85028
Principal Investigator: Kumaraswamy Sivakumar, MD
Phone: 480-314-1007
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Portland, Oregon 97239
Principal Investigator: Erika Finanger, MD MS
Phone: 971-544-3377
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Rochester, New York 14642
Principal Investigator: Emma Ciafaloni, MD
Phone: 585-275-4339
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2315 Stockton Blvd.
Sacramento, California 95817
(916) 734-2011
Principal Investigator: Craig McDonald, MD
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Saint Louis, Missouri 63110
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San Diego, California 92130
Principal Investigator: Carla Grosmann, MD
Phone: 858-966-6246
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Susan Apkon, MD
Phone: 206-884-2756
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Stanford, California 94305
Principal Investigator: John Day, MD
Phone: 650-206-3178
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Washington, District of Columbia 20010
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