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.
GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term study will be the same that the subject was receiving at the end of the previous DMD GIVINOSTAT study. As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according the rules detailed in the study protocol section 11.2.2.1. In addition, in case a subject will have a consistent (e.g., at least 2 consecutive evaluations) platelets count ≤150 x 10\^9/L and not meet the stopping criteria for platelets, the Investigator will have to reduce the dose of 1/3 or 20% less of the current dose as described in the study protocol section 10.5.1.3. During the first month of treatment, platelets count assessment will be performed weekly, while during the second month it will be performed every 2 weeks, in order to strictly monitor this parameter for safety reasons, with the exclusion of subjects coming from study DSC/11/2357/43 for which the first visit will be 4 months after the Visit 1/baseline visit. Study drug should be permanently interrupted if any of the following occurs: * severe drug-related diarrhoea (i.e., increase of ≥7 stools per day); * any drug-related SAE; * QTcF \>500 msec; * platelets count ≤50 x 10\^9/L; * white blood cells ≤2.0 x 10\^9/L; * hemoglobin ≤8.0 g/dL; To avoid laboratory errors and anomalous values, test must be confirmed with a repeated test performed on the next working day. The treatment should be stopped until the retest result becomes available. If the repeated test is still under the stopping limit value, study drug must be permanently discontinued. If the repeated test is acceptable, the subject can resume treatment. The Investigator will follow up the patient until resolution or acceptable stabilization of the event occurs and document all the relevant information, as applicable. After the resolution/stabilization of the event, the subject will be withdrawn from the study and the EOS Visit (see Section 12.1.10) will be performed. Any decision relevant to the dose adjustment and/or modification of schedule of assessments can be discussed with the Medical Monitor, but the final decision remains with the Investigator only or its authorized designee.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
206
suspension of givinostat (10 mg/mL)
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Type, incidence, and severity of treatment related/not related adverse events(AEs) and serious adverse event (SAEs)
Time frame: Through study completion, an average of 1 year
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation
Sacramento, California, United States
COMPLETEDRady Children's Hospital center - UCSD Department of Neuroscience
San Diego, California, United States
COMPLETEDConnecticut Children's Medical Center, Neurology Division
Hartford, Connecticut, United States
COMPLETEDChild Health Research Institute
Gainesville, Florida, United States
COMPLETEDMD Rare Disease Research, LLC
Atlanta, Georgia, United States
ENROLLING_BY_INVITATIONUniversity of Iowa Children's Hospital
Iowa City, Iowa, United States
ENROLLING_BY_INVITATIONWashington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111
St Louis, Missouri, United States
COMPLETEDShriners Hospitals for Children
Portland, Oregon, United States
COMPLETEDThe Children's Hospital of Philadelphia Colket Translational Research Building
Philadelphia, Pennsylvania, United States
COMPLETEDVirginia Commonwealth University Childrens Hospital of Richmond at
Richmond, Virginia, United States
COMPLETED...and 29 more locations