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Study of Ataluren in >=2 to <5 Year-Old Males With Duchenne Muscular Dystrophy
study id #: NCT02819557
condition: Duchenne Muscular Dystrophy
This is a Phase 2, multiple-dose, open-label study evaluating the safety, and pharmacokinetics (PK) of ataluren in patients aged >=2 to <5 years old with Duchenne muscular dystrophy caused by a nonsense mutation in the dystrophin gene. The study includes a 4-week screening period, a 4-week study period, and a 48-week extension period for patients who complete the 4-week study period (52 weeks total treatment).
mechanism of action: Stop codon read through to promote dystrophin production
start date: June 16, 2016
estimated completion: February 9, 2018
phase of development: Phase 2
size / enrollment: 14
This protocol describes a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in patients aged >=2 to <5 years old with nmDMD. In nmDMD, early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. The study will include a 4-week screening period, a 4-week study period, and a 48-week extension period for patients who complete the 4-week study period (52 weeks total treatment). The objective of the extension period is to assess the long-term safety of chronic administration of ataluren in this patient population.
- Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [Time Frame: 56 weeks]
Safety of ataluren as measured by type, frequency, severity, timing, and relationship to study drug of treatment emergent adverse events,laboratory abnormalities and electrocardiograms
- Maximum Plasma Concentration [Cmax] [Time Frame: 4 weeks]
Pharmacokinetic parameters (tmax, Cmax) based on frequent blood sampling for PK (drug concentrations) on Days 1 and 28 of ataluren treatment.
- Area Under the Curve [AUC] and Cmin [Time Frame: 4 weeks]
Pharmacokinetic parameters C(trough) @ 6hrs, and AUC (0-t), t=last time point) based on frequent blood sampling for PK (drug concentrations) on Days 1 and 28 of ataluren treatment.
- Descriptive assessments of efficacy [Time Frame: 56 weeks]
TFTs (time to walk/run 10 meters, time to climb 4 stairs, time to descend 4 stairs, and time to stand up from a supine position) ∙ NSAA ∙ Bayley-III (in patients <=30 months old)
- Effects on growth parameters [Time Frame: 56 weeks]
Changes in body weight, height, and BMI
• Males >=2 to <5 years of age
• Body weight >=12 kg
• Diagnosis of DMD
• Nonsense mutation in at least 1 allele of the dystrophin gene
• Participation in any other drug or device clinical investigation
• Ongoing use of prohibited concomitant medications
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