Study of Ataluren for Previously Treated Patients With nmDBMD in the US
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Purpose
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DBMD in approximately 10-15% of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is an open-label trial for patients with nonsense mutation dystrophinopathy who received ataluren in a prior PTC-sponsored study at a US clinical trial site. This trial will be conducted at sites in the US and will evaluate the long-term safety of ataluren, as determined by adverse events and laboratory abnormalities.
| Condition | Intervention | Phase |
|---|---|---|
|
Duchenne Muscular Dystrophy Becker Muscular Dystrophy |
Drug: Ataluren |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | An Open-label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy |
- Safety and Tolerability [ Time Frame: Open ] [ Designated as safety issue: Yes ]The safety profile of ataluren will be characterized by type, frequency, severity, timing, and relationship to ataluren of any adverse events or laboratory abnormalities.
| Estimated Enrollment: | 110 |
| Study Start Date: | November 2010 |
| Estimated Study Completion Date: | October 2012 |
| Estimated Primary Completion Date: | July 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Ataluren |
Drug: Ataluren
Oral powder for suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20mg/kg in the evening).
|
Detailed Description:
The study will enroll boys with nonsense mutation DBMD who have a history of exposure to ataluren in a prior PTC study in nmDBMD (PTC124-GD-004-DMD, PTC124-GD-004e-DMD, PTC124-GD-007-DMD, PTC124-GD-007e, and PTC124-GD-008-DMD) at a trial site in the US. Patients will receive study drug 3 times per day (at breakfast, lunch, and dinner). Study assessments will be performed at clinic visits during screening and every 12 weeks until the end of the study. Patients must also return to the clinic for a post treatment visit 6 weeks after the last dose of ataluren.
Eligibility| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial
- History of exposure to ataluren in a prior PTC study in nmDBMD at a trial site in the US
- Male sex
- Confirmed screening laboratory values within the specified ranges
- In subjects 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
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
- Known hypersensitivity to any of the ingredients or excipients of study drug
- Ongoing use of the following medications: coumarin based anti-coagulants, phenytoin, or tolbutamide, or systemic aminoglycoside therapy
- Ongoing uncontrolled medical/surgical condition, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the patient or make it unlikely that follow-up would be completed
Contacts and Locations| Contact: Diane Goetz | 866-282-5873 | patientinfo@ptcbio.com |
| United States, California | |
| University of California-Davis | Recruiting |
| Sacramento, California, United States | |
| United States, Colorado | |
| The Children's Hospital | Recruiting |
| Aurora, Colorado, United States | |
| United States, Florida | |
| Child Neurology Center of NW Florida | Recruiting |
| Gulf Breeze, Florida, United States, 32561 | |
| United States, Iowa | |
| University of Iowa Children's Hospital | Recruiting |
| Iowa City, Iowa, United States, 52242 | |
| United States, Kansas | |
| University of Kansas Medical Center | Recruiting |
| Kansas City, Kansas, United States, 66160 | |
| United States, Massachusetts | |
| Children's Hospital of Boston/Harvard Medical School | Recruiting |
| Boston, Massachusetts, United States | |
| United States, Minnesota | |
| University of Minnesota | Recruiting |
| Minneapolis, Minnesota, United States, 55455 | |
| United States, Missouri | |
| Washington University Medical School | Recruiting |
| St. Louis, Missouri, United States, 63110 | |
| United States, New York | |
| Columbia University Medical School | Recruiting |
| New York City, New York, United States | |
| United States, North Carolina | |
| Duke University Medical Center | Recruiting |
| Durham, North Carolina, United States, 27710 | |
| United States, Oregon | |
| Shriners Hospital for Children-Portland | Recruiting |
| Portland, Oregon, United States | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| United States, Texas | |
| Southwestern University | Recruiting |
| Dallas, Texas, United States | |
| United States, Utah | |
| University of Utah | Recruiting |
| Salt Lake City, Utah, United States, 84112 | |
| Study Director: | Jay Barth, MD | PTC Therapeutics, Inc. |
More Information
Additional Information:
Publications:
| Responsible Party: | Jay Barth, MD, Vice President, PTC Therapeutics, Inc |
| ClinicalTrials.gov Identifier: | NCT01247207 History of Changes |
| Other Study ID Numbers: | PTC124-GD-016 DMD |
| Study First Received: | November 22, 2010 |
| Last Updated: | August 16, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by PTC Therapeutics:
|
Duchenne muscular dystrophy Becker muscular dystrophy Nonsense mutation Premature stop codon DMD |
BMD nmDBMD DBMD Ataluren PTC124 |
Additional relevant MeSH terms:
|
Muscular Dystrophy, Duchenne Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 23, 2013