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A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From >=6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
study id #: NCT04336826
condition: Duchenne Muscular Dystrophy
status: not yet recruitingpurpose:
This study is designed to evaluate safety, tolerability, physical and motor development, and pharmacokinetics (PK) in children aged >=6 months to <2 years treated daily for 52 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
mechanism of action: Stop codon read through to promote dystrophin production
last updated: April 21, 2020
start date: June 1, 2020
estimated completion: June 20, 2023
phase of development: Phase 2
size / enrollment: 6
- Number of Participants With Treatment-Emergent Adverse Events [ Time Frame: Baseline up to Week 56 ]
- Pharmacokinetics: Area Under the Concentration Curve (AUC0-24) [ Time Frame: Day 1, Week 4, and at the end of study (Week 52) or early termination, whichever is first ]
- Pharmacokinetics: Maximum Concentration (Cmax) [ Time Frame: Day 1, Week 4, and at the end of study (Week 52) or early termination, whichever is first ]
- Pharmacokinetics: Time to Maximal Plasma Concentration (Tmax) [ Time Frame: Day 1, Week 4, and at the end of study (Week 52) or early termination, whichever is first ]
- Pharmacokinetics: Half-Life (t1/2) [ Time Frame: Day 1, Week 4, and at the end of study (Week 52) or early termination, whichever is first ]
• Eligible Sexes: male
• No clinically significant abnormality based upon laboratory assessments during the screening period, in the opinion of the Investigator; good general health, as determined during the screening period by medical history and physical examination (including vital sign measurements).
• Diagnosis of duchenne muscular dystrophy (DMD) based on an elevated serum creatine kinase and genotypic evidence of dystrophinopathy.
• Documentation of the presence of a nonsense mutation of the dystrophin gene.
• Verification that a blood sample was drawn for sequencing of the dystrophin gene.
• Participation in any drug investigation or received an investigational drug within three months prior to the Screening Visit or who anticipate participating in any other drug or device clinical investigation or receiving any other investigational drug within the duration of this study.
• Expectation of a major surgical procedure during the study period.
• Known hypersensitivity to any of the ingredients or excipients of the study drug (polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate).
• Prior and concomitant use of corticosteroids.
• Ongoing use of the following drugs:
- Systemic aminoglycoside therapy and/or intravenous (IV) vancomycin.
- Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel.
- Inducers of UGT1A9 (for example, rifampicin), or substrates of OAT1 or OAT3 (for example, ciprofloxacin, adefovir, oseltamivir, aciclovir, captopril, furosemide, bumetanide, valsartan, pravastatin, rosuvastatin, atorvastatin, pitavastatin).
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