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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, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ataluren | Experimental | Participants will receive 3 doses of ataluren oral suspension per day (10 mg/kg in the morning, 10 mg/kg at mid-day and 20 mg/kg in the evening). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ataluren | Drug | Ataluren will be administered as per the dose and schedule specified in the arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events | Baseline up to end of study (up to approximately 8 years) | |
| Number of Participants With Laboratory Parameters Abnormalities | Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data. | Baseline up to end of study (up to approximately 8 years) |
| Number of Participants With Abnormal Physical Findings | Physical findings include weight, physical examination data, systolic and diastolic blood pressure, radial pulse rate, and body temperature. | Baseline up to end of study (up to approximately 8 years) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Vinay Penematsa, MD | PTC Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Childrens Hospital | Phoenix | Arizona | 85016 | United States | ||
| Children's Hospital of Los Angeles |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 17389552 | Result | Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. | |
| 17450125 |
| Label | URL |
|---|---|
| PTC Therapeutics, Inc website | View source |
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| Los Angeles |
| California |
| 90027 |
| United States |
| UCLA | Los Angeles | California | 90095 | United States |
| Stanford University | Palo Alto | California | 94304 | United States |
| University of California-Davis | Sacramento | California | 95817 | United States |
| University of CA, San Francisco-Benioff Children's Hospital | San Francisco | California | 94158 | United States |
| Children's Hospital Colorado - Center for Cancer and Blood Disorders | Aurora | Colorado | 80045 | United States |
| Child Neurology Center of NW Florida NW | Gulf Breeze | Florida | 32561 | United States |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | United States |
| Rush Univ Medical Center | Chicago | Illinois | 60612 | United States |
| University of Iowa Children's Hospital | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Children's Hospital of Boston/Harvard Medical School | Boston | Massachusetts | 02115 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Washington University Medical School | St Louis | Missouri | 63110 | United States |
| Columbia University Pediatric Neuromuscular Center | New York | New York | 10032 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Cincinnati Childrens Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Shriners Hospital for Children-Portland | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 16148 | United States |
| Childrens Medical Center Dallas | Dallas | Texas | 75207 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Texas Health Science Center at San Antonio | San Antonio | Texas | 78229 | United States |
| University of Utah | Salt Lake City | Utah | 84112 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Alberta Children's Hospital | Calgary | Alberta | Canada |
| British Columbia Children's Hospital | Vancouver | British Columbia | Canada |
| London Health Sciences Centre | London | Ontario | Canada |
| Children's Hospital of East Ontario | Ottawa | Ontario | Canada |
| CHU de Québec - Université Laval | Québec | Canada |
| Result |
| Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. |
| Protocol V7.0 | View source |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C515878 | ataluren |
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