This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
* Planned screening duration: approximately 4 weeks * Planned Core Treatment duration: approximately 48 weeks * Planned Extension Treatment duration: approximately 96 weeks * Planned Follow Up duration: approximately 4 weeks (± 7 days) * Total duration of study participation: up to 151 weeks (ie, 37-38 months)
Male children aged ≥2 to <6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study)
Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)
A genetic diagnosis of DMD
Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit
Core Phase
Exposure to another investigational drug within 3 months prior to the start of the study drug
Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug
Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug
Italfarmaco
Lead sponsor
Fortrea
Collaborator