Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

Exclusion Criteria

• •1. Exposure to another investigational drug within 3 months prior to the start of the study drug
• •2. Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug
• •3. Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug
• •4. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of the study drug (eg, growth hormone). Note: Vitamin D, calcium, and any other supplements will be allowed.
• •5. Have had surgery that might have an effect on muscle strength or function within 3 months prior to start of the study drug or planned surgery at any time during the study
• •6. The presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect subject's safety, making it unlikely to complete the study or to be compliant with study-specific requirements that could impair the assessment of study results
• •7. Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator clinical medical judgement
• •8. Platelet count, white blood cells, and/or haemoglobin counts \< lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results \[\<LLN\], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \<LLN, the subject will be excluded)
• •9. Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, \>1.5 × upper limit of normal \[ULN\]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease
• •10. Inadequate renal function, as defined by serum Cystatin C result \>2 × ULN (Note: if the value is \>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 × ULN, the subject will be excluded)
• •11. Fasting triglycerides \>300 mg/dL (3.42 mmol/L) at screening (Note: if the value is \>300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still \>300 mg/dL in fasting, the subject should be excluded)
• •12. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening
• •13. Baseline corrected QT interval using Fridericia's formula (QTcF) \>450 msec (as the mean of 3 consecutive readings taken 5 minutes apart) or history of additional risk factors for torsades de pointes (ie, heart failure, hypokalaemia, or family history of long QT syndrome)
• •14. Psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator's clinical medical judgement
• •15. Hypersensitivity to any component of study drug
• •16. Sorbitol intolerance or malabsorption or have the hereditary form of fructose intolerance.
• •17. Body weight \<10 kg at screening.
• •1. Platelet count, white blood cells, and/or haemoglobin \<LLN at EOT/V12 (Note: for abnormal laboratory test results \[\<LLN\], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \<LLN, the subject will be excluded)
• •2. Current liver disease or impairment, including but not limited to an elevated total bilirubin (ie, \>1.5 × ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert disease
• •3. Inadequate renal function, as defined by serum Cystatin C result \>2 × ULN (Note: if the value is \>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 × ULN, the subject will be excluded)
• •4. Fasting triglycerides \>300 mg/dL (3.42 mmol/L; Note: if the value is \>300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still \>300 mg/dL in fasting condition, the subject should be excluded)
• •5. Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results
• •6. Evidence of psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator's clinical medical judgement.