Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
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|ClinicalTrials.gov Identifier: NCT03373968|
Recruitment Status : Enrolling by invitation
First Posted : December 14, 2017
Last Update Posted : April 28, 2021
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Givinostat||Phase 2 Phase 3|
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 220.127.116.11.
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 After the resolution/stabilization of the event, the subject will be withdrawn from the study and the EOS Visit (see Section 12.1.10) will be performed.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 (Clinical Trial)|
|Estimated Enrollment :||156 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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|
|Actual Study Start Date :||October 24, 2017|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2023|
Givinostat oral suspension (10 mg/mL) twice daily in a fed state
suspension of givinostat (10 mg/mL)
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through study completion, an average of 1 year ]Type, incidence, and severity of treatment related/not related adverse events(AEs) and serious adverse event (SAEs)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03373968