Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
- Registration Number
- NCT01247207
- Lead Sponsor
- PTC Therapeutics
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 270
- 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 and effected nmDBMD siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).
- Fertile men, who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 50 days after the last dose of study drug.
- Willingness and ability to comply with scheduled visits, drug administration and return plan, study procedures, laboratory tests, and study restrictions Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.
- 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 for ataluren naïve participants (siblings) or participants who have a temporary treatment gap of 1 year before entering study
- Known hypersensitivity to any of the ingredients or excipients of ataluren (refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate).
- Ongoing intravenous (IV) aminoglycoside or IV vancomycin 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Ataluren Ataluren 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).
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events Baseline up to end of study (up to approximately 8 years) Number of Participants With Laboratory Parameters Abnormalities Baseline up to end of study (up to approximately 8 years) Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data.
Number of Participants With Abnormal Physical Findings Baseline up to end of study (up to approximately 8 years) Physical findings include weight, physical examination data, systolic and diastolic blood pressure, radial pulse rate, and body temperature.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (34)
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Rush Univ Medical Center
🇺🇸Chicago, Illinois, United States
University of Iowa Children's Hospital
🇺🇸Iowa City, Iowa, United States
Child Neurology Center of NW Florida NW
🇺🇸Gulf Breeze, Florida, United States
Phoenix Childrens Hospital
🇺🇸Phoenix, Arizona, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
UCLA
🇺🇸Los Angeles, California, United States
Stanford University
🇺🇸Palo Alto, California, United States
University of California-Davis
🇺🇸Sacramento, California, United States
University of CA, San Francisco-Benioff Children's Hospital
🇺🇸San Francisco, California, United States
Children's Hospital Colorado - Center for Cancer and Blood Disorders
🇺🇸Aurora, Colorado, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Children's Hospital of Boston/Harvard Medical School
🇺🇸Boston, Massachusetts, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Washington University Medical School
🇺🇸Saint Louis, Missouri, United States
Columbia University Pediatric Neuromuscular Center
🇺🇸New York, New York, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Cincinnati Childrens Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Shriners Hospital for Children-Portland
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
Childrens Medical Center Dallas
🇺🇸Dallas, Texas, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
University of Texas Health Science Center at San Antonio
🇺🇸San Antonio, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, 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
🇨🇦Quebec, Canada