Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)



Status:Enrolling by invitation
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:Any
Updated:3/22/2019
Start Date:November 30, 2010
End Date:December 31, 2020

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An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams
per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren
in a PTC sponsored clinical trial or treatment plan.

The treatment will continue under this protocol until consent withdrawal by participants,
withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by
investigator, withdrawal due to participant unable to tolerate ataluren, participant is
eligible to participate in another ataluren nmDBMD clinical trial program initiated by
sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until
ataluren becomes commercially available.


Inclusion Criteria:

- Evidence of signed and dated informed consent/assent document(s) indicating that the
participant (and/or his parent/legal guardian) has been informed of all pertinent
aspects of the trial. Note: If the study candidate is considered a child under local
regulation, a parent or legal guardian must provide written consent prior to
initiation of study screening procedures and the study candidate may be required to
provide written assent. The rules of the responsible Institutional Review
Board/Independent Ethic Committee (IRB/IEC) regarding whether one or both parents must
provide consent and the appropriate ages for obtaining consent and assent from the
participant should be followed.

- History of exposure to ataluren in a prior PTC study or treatment plan in nmDBMD.

- In participants who are sexually active, willingness to abstain from sexual
intercourse or employ a barrier or medical method of contraception during ataluren
administration and the 6-week follow up period.

- Willingness and ability to comply with scheduled visits, drug administration plan,
study procedures, laboratory tests, and study restrictions Note: Psychological,
social, familial, or geographical factors that might preclude adequate study
participation should be considered.

Exclusion Criteria:

- Exposure to another investigational drug within 1 month prior to start of study
treatment.

- Eligibility for another ataluren clinical trial that is actively enrolling study
participants.

- Positive for Hepatitis B core antibody or Hepatitis C antibody at screening.

- Known hypersensitivity to any of the ingredients or excipients of ataluren (Litesse®
UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127
[poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla,
Cab-O-Sil® M5P [colloidal silica], magnesium stearate).

- Ongoing use of the systemic aminoglycoside therapy.

- Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or
laboratory abnormality that, in the investigator's opinion, could adversely affect the
safety of the participant or make it unlikely that follow-up would be completed.
We found this trial at
23
sites
Aurora, Colorado 80045
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
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
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Minneapolis, Minnesota 55455
(612) 625-5000
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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Minneapolis, MN
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
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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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
University of Utah Research is a major component in the life of the U benefiting...
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Boston, MA
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Calgary, Alberta
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Charlottesville, Virginia 22903
(434) 924-0311
University of Virginia The University of Virginia is distinctive among institutions of higher education. Founded...
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1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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Dallas, Texas 75225
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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Gulf Breeze, Florida 32561
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6621 Fannin St
Houston, Texas 77030
(832) 824-1000
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, IA
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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Los Angeles, California 90095
(310) 825-4321
UCLA UCLA's primary purpose as a public research university is the creation, dissemination, preservation and...
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Pittsburgh, Pennsylvania 16148
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1 Shields Ave
Sacramento, California 95616
(530) 752-1011
University of California-Davis As we begin our second century, UC Davis is poised to become...
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Saint Louis, Missouri 63110
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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