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enrolling by invitation

Study of ataluren in previously treated participants with nonsense mutation dystrophinopathy (nmDBMD)

key information

study id #: NCT01247207

condition: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

status: enrolling by invitation


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.

intervention: Ataluren

mechanism of action: Stop codon read through to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01247207

last updated: July 19, 2019

study details

start date: November 30, 2010

estimated completion: December 31, 2020

phase of development: Phase 3

size / enrollment: 160

primary outcomes:

  • Number of Participants With Adverse Events [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]
  • Number of Participants With Laboratory Parameters Abnormalities [ Time Frame: 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 [ Time Frame: 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.

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.

study contacts

sponsor: PTC Therapeutics

investigators: Francesco Bibbiani, MD

locations: United States, Canada