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|
| Study Name: |
Ataluren (PTC 124) Open Label Extension Study in Cystic Fibrosis |
| Study Type: |
Interventional |
| Intervention Category: |
CFTR Modulation |
| Study Sponsor: |
PTC |
| Study Phase: |
3 |
| Recruitment Status: |
Recruiting Completed |
| Study Drug(s): |
PTC124 |
| Number of Participants Being Recruited: |
190 |
| Single / Multi-Center: |
Multi-Center |
| STUDY BACKGROUND INFORMATION: |
| Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of subjects with the disease. Ataluren (PTC124) is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren (PTC124) in adult and pediatric patients with nonsense mutation CF, as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology. |
| Age: |
>= 6 Years
|
| FEV1: |
40 - 90 Percent Predicted
|
| P. aeruginosa status: |
Not applicable |
| B. cepacia status: |
Not applicable |
Other Primary Eligibility Requirements:
To be included in this study, subjects must have completed the placebo controlled phase 3 study of PTC (PTC124-009) and as noted for that study must have a documented nonsense mutation in at least 1 allele of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by gene sequencing.
|
| Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below. |
| Sponsor Contact Information: |
Goetz, Diane
(908) 912-9256
dgoetz@ptcbio.com
|
| PARTICIPATING SITES: |
| The sites listed below are currently recruiting subjects. Please note that this list generally lists either the pediatric or adult program but not both even though both programs may be recruiting subjects. Also, this list does not include sites that may be participating in the study but are not yet open for recruitment. If you are interested in this study please contact your center for more information. |
| AL |
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The Children's Hospital/University of Alabama at Birmingham (Pediatric), Birmingham, AL, 35233
Contact Information:
Hathorne, Heather
Phone: (205) 638-9568
Email: hhathorne@peds.uab.edu
|
| CA |
-
Miller Children's Hospital (Pediatric), Long Beach, CA, 90801
Contact Information:
O'Donnell, Nan
Phone: (562) 933-5607
Email: nodonnell@memorialcare.org
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Stanford University Medical Center (Pediatric), Palo Alto, CA, 94304
Contact Information:
Dunn, Colleen
Phone: (650) 736-0388
Email: cedunn@stanford.edu
|
| CO |
|
|
| GA |
-
Emory University (Pediatric), Atlanta, GA, 30322
Contact Information:
Hunter, Eric
Phone: (404) 727-9960
Email: eahunte@emory.edu
|
| IL |
-
Ann and Robert H. Lurie Children's Hospital of Chicago (Pediatric), Chicago, IL, 60611
Contact Information:
Singh, Noopur
Phone: (312) 227-6861
Email: nosingh@luriechildrens.org
|
| MA |
|
|
| MD |
-
Johns Hopkins University (Pediatric), Baltimore, MD, 21287
Contact Information:
Callahan, Karen
Phone: (443) 287-8983
Email: kcallah1@jhmi.edu
|
| MO |
-
St. Louis Children's Hospital Washington University (Pediatric), St. Louis, MO, 63110
Contact Information:
Rodgers, Denise
Phone: (314) 286-1176
Email: rodgers@kids.wustl.edu
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St. Louis Children's Hospital Washington University (Pediatric), St. Louis, MO, 63110
Contact Information:
Burks, Patricia
Phone: (314) 454-5175
Email: Burks_P@kids.wustl.edu
|
| NC |
-
University of North Carolina at Chapel Hill (Pediatric), Chapel Hill, NC, 27599
Contact Information:
Stephenson, Kelly
Phone: (919) 843-7121
Email: kelly.stephenson@unc.edu
|
| NY |
-
Beth Israel Medical Center (Pediatric), New York, NY, 10003
Contact Information:
Grece, Carroll Anne
Phone: (212) 420-4196
Email: cgrece@chpnet.org
|
| OH |
-
University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital (Pediatric), Cleveland, OH, 44106
Contact Information:
Ksenich, Roberta (Bobbi)
Phone: (216) 844-5733
Email: Roberta.Ksenich@UHHospitals.org
|
| PA |
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University of Pittsburgh School of Medicine (Adult), Pittsburgh, PA, 15213
Contact Information:
Hartigan, Elizabeth
Phone: (412) 692-7060
Email: elizabeth.hartigan@chp.edu
|
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