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)
Completed · Phase 2
Conditions studied: Nonsene Mutation Duchenne Muscular Dystrophy
In brief
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to \<2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
Key facts
- Study ID
- NCT04336826
- Run by
- PTC Therapeutics
- People needed
- 6
- Starts
- 2021-12-29
- Expected to finish
- 2023-08-07
- Last updated by the study team
- 2024-04-01
Who can join
Age: 1 and older, up to 2. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- Body weight ≥7.5 kilograms (kg)
- 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 as determined by gene sequencing prior to enrollment.
You may not qualify if…
- Participation in any drug or device investigation or whose sibling is currently participating in a blinded portion of another ataluren study 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).
- 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).
Where it is running
- Rare Disease Research, LLC — Atlanta, Georgia, United States
Full record on ClinicalTrials.gov
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