Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy

Status:	Completed
Conditions:	Neurology, Orthopedic
Therapuetic Areas:	Neurology, Orthopedics / Podiatry
Healthy:	No
Age Range:	2 - 5
Updated:	1/30/2019
Start Date:	June 16, 2016
End Date:	February 9, 2018

A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy

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).

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.

Inclusion Criteria:

- 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

Exclusion Criteria:

- Participation in any other drug or device clinical investigation

- Ongoing use of prohibited concomitant medications

We found this trial at

sites

Cincinnati Children's Hospital Medical Center

3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229

1-513-636-4200