Nonsene Mutation Duchenne Muscular Dystrophy Clinical Trial
Official title:
An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy
| Verified date | February 2024 |
| Source | PTC Therapeutics |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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).
| Status | Completed |
| Enrollment | 6 |
| Est. completion date | August 7, 2023 |
| Est. primary completion date | August 7, 2023 |
| Accepts healthy volunteers | No |
| Gender | Male |
| Age group | 6 Months to 2 Years |
| Eligibility | 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: 1. Systemic aminoglycoside therapy and/or intravenous (IV) vancomycin. 2. Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel. 3. 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). |
| Country | Name | City | State |
|---|---|---|---|
| United States | Rare Disease Research, LLC | Atlanta | Georgia |
| Lead Sponsor | Collaborator |
|---|---|
| PTC Therapeutics |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. Serious adverse event (SAE): an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event. A TEAE was defined as an AE that occurred or worsened while on ataluren (on or after first dose of ataluren) up to 4 weeks after the last dose. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Baseline up to Week 28 | |
| Secondary | Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren | Predose up to 12 hours postdose at Week 24 | ||
| Secondary | Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-t) of Ataluren | Predose up to 12 hours postdose at Week 24 | ||
| Secondary | Maximum Concentration (Cmax) of Ataluren | Predose up to 12 hours postdose at Week 24 | ||
| Secondary | Time to Maximum Plasma Concentration (Tmax) of Ataluren | Predose up to 12 hours postdose at Week 24 | ||
| Secondary | Trough Concentration (Ctrough) of Ataluren | Predose up to 12 hours postdose at Week 24 |