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
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
• 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 inoff-target group,
⁃ never been randomized because, the enrollment in the off target group was completed.
• Aged ≥6 years old;
• Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);
• 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-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly.