Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT02851797|
Recruitment Status : Recruiting
First Posted : August 2, 2016
Last Update Posted : July 16, 2018
it is a randomised, double blind, parallel group, placebo controlled study. A total of 213 male ambulant subjects will be randomised 2:1 (givinostat:placebo).
Subjects will be stratified for their concomitant use of steroids in 4 strata:
- Deflazacort daily regimen
- Deflazacort intermittent regimen
- Other steroids daily regimen
- Other steroids intermittent regimen. The study duration is planned for 19 months.
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: givinostat Drug: placebo||Phase 3|
Givinostat or placebo oral suspension (10 mg/mL) will be administered orally as 2 oral doses daily while the subject is in fed state, according to the child's weight.
Study drug should be permanently stopped if any of the following occur:
- severe drug-related diarrhoea;
- any drug-related Serious Adverse Event;
- QTcF >500 msec;
- platelets count ≤50 x 109/L.
Study drug should be temporarily stopped if any of the following occur:
- platelets count <75 x 109/L but >50 x 109/L (the treatment should be temporarily stopped and a platelets count has to be performed and re-tested until platelets will be normalized);
- moderate or severe diarrhoea.
In case the study drug was temporarily stopped, the study drug can be resumed at a level 1/3 smaller than the one at which the Adverse Event leading to temporary stop occurred, once platelets are normalized or diarrhoea is mild (if treatment was stopped for moderate or severe diarrhoea).
Two interim analyses are planned and will be conducted by the IDMC in order to ensure study integrity.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||213 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||June 1, 2017|
|Estimated Primary Completion Date :||June 2020|
|Estimated Study Completion Date :||June 2020|
Active Comparator: givinostat
Givinostat oral suspension (10 mg/mL) twice daily in a fed state
suspension of givinostat (10 mg/mL)
Placebo Comparator: placebo
Placebo oral suspension (10 mg/mL) twice daily in a fed state
suspension manufactured to mimic givinostat
- mean change in 4 standard stairs climb [ Time Frame: 18 months ]the primary endpoint is the mean change in 4 standard stairs climb test results before and after 18 months of treatment of givinostat versus placebo
- Other functional test as 6MWT [ Time Frame: 18 months ]the mean change in 6MWT
- time to rise from floor [ Time Frame: 18 months ]the mean change in time to rise from floor
- Magnetic Resonance Spetroscopy [ Time Frame: 18 months ]the mean change in fat fraction of vastus lateralis muscles at MRS
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): NCT02851797
|Contact: Reference Study ID Number EPYDIS (DSC/14/2357/48)||+39 026443 ext firstname.lastname@example.org|
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