A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
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
• Corticosteroid treatment considerations:
‣ For subject receiving a stable dose or oral systemic corticosteroids:
‣ No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 3 months immediately prior to the start of the study drug or
⁃ For subjects without current corticosteroid treatment:
∙ Must not start corticosteroids in the Core Phase of the study (ie, first 48 weeks).
• Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit
• Give informed consent and /or assent in writing signed by the parent/legal guardian and/or subject (according to local regulation)
• In stable oral systemic corticosteroids treatment with no significant change in dose or dosing regimen (except for adjustments due to body weight change). For subjects without corticosteroids during the Core Phase, the treatment can be started based on the Investigator's clinical medical judgement.