Phase 2
Completed N=6
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)
Nonsene Mutation Duchenne Muscular Dystrophy
Source: ClinicalTrials.gov NCT04336826 ↗
Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Apr 2024
Primary outcomePrimary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 5 Participants
Summary
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
5 | — |
| SECONDARY Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren |
167 | — |
| SECONDARY Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren |
NA | — |
| SECONDARY Maximum Concentration (Cmax) of Ataluren |
12.6 | — |
| SECONDARY Time to Maximum Plasma Concentration (Tmax) of Ataluren |
2.08 | — |
| SECONDARY Trough Concentration (Ctrough) of Ataluren |
3.48 | — |
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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).
Data sourced from ClinicalTrials.gov (NCT04336826). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.