Study of SRP-4045 and SRP-4053 in DMD Patients



Status:Recruiting
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:7 - 13
Updated:3/15/2019
Start Date:September 28, 2016
End Date:May 2023
Contact:Medical Information
Email:clinicaltrials@sarepta.com
Phone:+1 888 727 3782

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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

The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053
compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion
mutations amenable to skipping exon 45 and exon 53, respectively.

This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and
safety of SRP-4045 and SRP-4053. Eligible patients 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 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 patients will receive open-label
active treatment for 48 weeks (up to Week 144 of study).

The study will enroll approximately 222 patients, with a planned minimum target of 111
patients amenable to exon 45 skipping and 111 patients amenable to exon 53 skipping.

Approximately 148 patients will be randomized to receive active treatment with either
SRP-4045 or SRP-4053 (depending on deletion mutation), and 74 patients will be randomized to
receive placebo. Twice as many patients will 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 six-minute walk test (6MWT). All patients 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.

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

- Intact right and left biceps or 2 alternative upper muscle groups

- Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters

- Stable pulmonary and cardiac function: forced vital capacity (FVC) equal to or greater
than 50% predicted and left ventricular ejection fraction (LVEF) greater than 50%

Exclusion Criteria:

- Previous treatment with SMT C1100 (BMN-195) at any time

- Treatment with gene therapy at any time

- Previous treatment with PRO045 or PRO053 within 24 weeks prior to Week 1

- Current or previous treatment with any other experimental treatment (other than
deflazacort) within 12 weeks prior to Week 1

- Participation in any other DMD interventional clinical study within 12 weeks prior to
Week 1

- Major surgery within 3 months prior to Week 1

- Presence of other clinically significant illness

- Major change in physical therapy regimen within 3 months prior to Week 1
We found this trial at
29
sites
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Jerry R Mendell, MD
Phone: 614-722-8528
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Barry Byrne, MD, PhD
Phone: 352-294-8705
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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Gainesville, FL
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Gulf Breeze, Florida 32561
Principal Investigator: Ben Renfroe, MD
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282 Washington St
Hartford, Connecticut 06106
(860) 545-9000
Principal Investigator: Gyula Acsadi, MD, PhD
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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Hartford, CT
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Leigh Maria Ramos-Platt, MD
Phone: 323-361-5825
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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601 Children's Lane
Norfolk, Virginia 23507
(757) 668-7000
Principal Investigator: Crystal Proud, MD
Phone: 757-668-9356
Children's Hospital of The King's Daughters Children
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Norfolk, VA
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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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Philadelphia, PA
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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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Sacramento, California 95814
Principal Investigator: Craig McDonald, MD
Phone: 916-734-0968
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Russell Butterfield, MD, PhD
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
Principal Investigator: Han Phan, MD
Phone: 678-883-6897
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Basil Darras, MD
Phone: 857-218-4677
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Boston, MA
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Nancy Kuntz, MD
Phone: 312-227-4483
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Cincinnati, Ohio 45229
Principal Investigator: Chet Villa, MD
Phone: 513-803-9003
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1935 Medical District Dr
Dallas, Texas 75235
(214) 456-7000
Principal Investigator: Susan Iannaccone, MD
Phone: 214-456-2463
Children's Medical Center of Dallas Children's Medical Center is private, not-for-profit, and is the fifth-largest...
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Iowa City, Iowa 52242
Principal Investigator: Katherine Mathews, MD
Phone: 319-356-2673
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Jeffery Statland, MD
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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Las Vegas, Nevada 89145
Principal Investigator: Jonathan McKinnon, MD
Phone: 702-505-4230
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10833 Le Conte Ave
Los Angeles, California 90095
(310) 825-4321
Principal Investigator: Perry Shieh, MD, PhD
Phone: 310-825-3264
David Geffen School of Medicine, UCLA In 2002 Mr. David Geffen announced a $200 million...
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Principal Investigator: Matthew Harmelink, MD
Phone: 414-266-6792
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Parkville, Victoria
Principal Investigator: Monique Ryan
Phone: ??+61399366157
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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: 503-221-3471
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Rochester, New York 14642
Principal Investigator: Emma Ciafaloni, MD
Phone: 585-275-4339
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Saint Louis, Missouri 63110
Principal Investigator: Ann M Connolly, MD
Phone: 314-362-6991
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3020 Children's Way
San Diego, California 92123
Principal Investigator: Carla Grosmann, MD
Phone: 858-966-8208
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Stanford, California 94305
Principal Investigator: John Day, MD
Phone: 650-206-3178
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