Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy



Status:Recruiting
Conditions:Neurology, Neurology, Neurology, Neurology, Neurology, Women's Studies
Therapuetic Areas:Neurology, Reproductive
Healthy:No
Age Range:5 - Any
Updated:3/22/2019
Start Date:July 6, 2017
End Date:December 31, 2021
Contact:Mary Frances Harmon
Email:mharmon@ptcbio.com
Phone:908-912-9256

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A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne
muscular dystrophy.

This study is a randomized, double-blind, placebo-controlled, 72-week study followed by a
72-week open-label period. The purpose is to characterize the long-term effects of
ataluren-mediated dystrophin restoration on disease progression. Participants will be
randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study
drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all
participants will receive open-label ataluren for an additional 72 weeks (144 weeks in
total). Study assessments will be performed at clinic visits every 12 weeks during the
double-blind period and every 24 weeks during the open-label period. The total sample size of
~250 subjects will include ~160 subjects who meet the criteria for inclusion in the primary
analysis population (age 7 to 16 years old, baseline six minute walk distance (6MWD) greater
than or equal to (>=) 300 meters, supine to stand >= 5 seconds). The study will be conducted
in the United States and other countries around the world.

Inclusion Criteria -

- Male sex.

- Age ≥5 years.

- Phenotypic evidence of Duchenne Muscular Dystrophy

- Nonsense point mutation in the dystrophin gene

- Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum
of 12 months immediately prior to start of study treatment, with no significant change
in dosage or dosing regimen for a minimum of 3 months immediately prior to start of
study treatment

- 6MWD ≥150 meters

- Ability to perform timed function tests within 30 seconds

- Willingness and ability to comply with scheduled visits, drug administration plan,
study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start
of study treatment.

- Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.

- Prior or ongoing therapy with ataluren.

- Known hypersensitivity to any of the ingredients or excipients of the study drug

- Exposure to another investigational drug within 6 months prior to start of study
treatment, or ongoing participation in any interventional clinical trial.

- History of major surgical procedure within 12 weeks prior to start of study treatment,
or expectation of major surgical procedure during the 72-week placebo-controlled
treatment period.

- Requirement for daytime ventilator assistance or any use of invasive mechanical
ventilation via tracheostomy.

- Uncontrolled clinical symptoms and signs of congestive heart failure

- Elevated serum creatinine or cystatin C at screening.
We found this trial at
28
sites
Westmead, New South Wales 2145
Principal Investigator: Kristi Jones, MD
Phone: +61 02 9845 0495
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: Cuixia Tian, MD
Phone: 513-803-9036
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Kevin Flanigan, 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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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Leigh 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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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Peter Karachunski, MD
Phone: 612-626-4690
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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630 W 168th St
New York, New York 10032
Principal Investigator: Darryl De Vivo, MD
Phone: 212-342-3679
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601 Children's Lane
Norfolk, Virginia 23507
(757) 668-7000
Principal Investigator: Crystal Proud, MD
Phone: 757-469-9123
Children's Hospital of The King's Daughters Children
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Russell Butterfield, MD
Phone: 801-587-1227
University of Utah Research is a major component in the life of the U benefiting...
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4502 Medical Drive
San Antonio, Texas 78284
(210) 567-7000
Principal Investigator: Ratna Bhavaraju-Sanka, MD
Phone: 210-567-8222
University of Texas Health Science Center at San Antonio The University of Texas Health Science...
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Ann Arbor, Michigan 48167
Principal Investigator: Erin Neil Knierbein, MD
Phone: 734-998-9972
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Boston, Massachusetts 02115
Principal Investigator: Partha Ghosh, MD
Phone: 617-355-7310
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3901 Beaubien St
Detroit, Michigan 48201
(313) 745-5437
Principal Investigator: Lalitha Sivaswamy, MD
Phone: 313-745-0627
Children's Hospital of Michigan Since 1886, the Children's Hospital of Michigan has been dedicated to...
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801 7th Avenue
Fort Worth, Texas 76104
(682) 885-4000
Principal Investigator: Warren Marks, MD
Phone: 682-885-1904
Cook Children's Medical Center Cook Children's Health Care System is a not-for-profit, nationally recognized pediatric...
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Green Bay, Wisconsin 54303
Principal Investigator: Terence Edgar, MD
Phone: 920-272-1044
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Gulf Breeze, Florida 32561
Principal Investigator: Weldon Mauney III, MD
Phone: 850-934-1299
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6621 Fannin St
Houston, Texas 77030
(832) 824-1000
Principal Investigator: Tim Lotze, MD
Phone: 832-822-1255
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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Indianapolis, Indiana 46202
Principal Investigator: Marcia Felker, MD
Phone: 317-274-4928
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Jeffrey Statland, MD
Phone: 913-945-9922
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Principal Investigator: Matthew Harmelink, MD
Phone: 414-266-3355
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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20 York St, N20 York St,
New Haven, Connecticut 06520
(203) 688-4242
Principal Investigator: Cristian Ionita, MD
Phone: 203-785-5977
Yale-New Haven Hospital Relying on the skill and expertise of more than 4,500 university and...
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Oakland, California 94143
Principal Investigator: Jonathan Strober, MD
Phone: 415-476-2905
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Palo Alto, California 94304
Principal Investigator: John Day, MD
Phone: 650-206-3178
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Saunder Bernes, MD
Phone: 602-933-0641
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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Pittsburgh, Pennsylvania 15261
Principal Investigator: Hoda Abdel-Hamid, MD
Phone: 412-692-5176
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Portland, Oregon 97239
Principal Investigator: Erika Finanger, MD
Phone: 971-282-4616
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Sacramento, California 95817
Principal Investigator: Craig McDonald, MD
Phone: 916-734-0384
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San Bernardino, California 92408
Principal Investigator: David Michelson, MD
Phone: 909-558-5830
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Fawn Leigh, MD
Phone: 206-987-5457
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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