Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past

Trial details

Who runs it

Italfarmaco S.p.A.

Trial code

EU 2023-504520-26-00

Record updated

2026. november 6.

Original description

To assess the long-term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program and with naïve GIVINOSTAT DMD subjects, i.e. subjects screened in study DSC/14/2357/48 who met: - all the inclusion criteria and none of the exclusion criteria, and - never been randomized because the enrollment in the off-target group was completed.Read full original descriptionShow less

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.

To assess the long-term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program and with naïve GIVINOSTAT DMD subjects, i.e. subjects screened in study DSC/14/2357/48 who met: - all the inclusion criteria and none of the exclusion criteria, and - never been randomized because the enrollment in the off-target group was completed.

Inclusion: Subjects must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met: all the inclusion criteria and none of the exclusion criteria, had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or >30%, i.e. included in "off-target" group, never been randomized because the enrollment in the off-target group was completed; Subjects aged ≥6 years; Subjects are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations); Subjects must be willing to use adequate contraception: Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following: -True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. -Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral, transdermal, injectable or implanted steroid-based contraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such as for example cervical cap with spermicide jelly.

Exclusion: Use of any current investigational drug other than Givinostat; Have a psychiatric illness/social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures; Have any hypersensitivity to the components of study medication; 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; Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD; Have heart failure (New York Heart Association Class III or IV); Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed; Have a baseline QTcF >450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome); Have platelets count, White Blood Cell and Hemoglobin at screening < Lower Limit of Normal (LLN) (for abnormal screening laboratory test results (; Have Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit* (for abnormal screening laboratory test results (>300mg/dl), the triglycerides will be repeated once; if the repeat test result is still >300mg/dl, then exclusionary; Have inadequate renal function, as defined by serum Cystatin C >2 x the upper limit of normal (ULN) at screening visit. If the value is >2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 x ULN, the subject should be excluded); Have a current liver disease or impairment, including but not limited to an elevated total bilirubin(i.e. > 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's; Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.

EU CTIS 2023-504520-26-00