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 : May 1, 2019
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.
- white blood cells ≤2.0 x 109/L
- hemoglobin ≤8.0 g/dL
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.
- white blood cell <3.0 x 109/L but >2.0 x 109/L (the treatment should be temporarily stopped and white blood cells have to be measured by 1 week and re-tested until white blood cells will be normalized);
- hemoglobin <10.0 g/dL but > 8.0 g/dL (the treatment should be temporarily stopped and hemoglobin has to be measured by 1 week and re-tested until hemoglobin will be normalized);
- Triglycerides >300 mg/dL (3.42 mmol/L) in fasting condition (the treatment should be temporarily stopped and triglycerides has to be measured every 2 weeks until triglycerides return to levels below 300mg/dL (3.42 mmol/L)
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 and/or white blood cell and/or hemoglobin are normalized and/or triglycerides return to levels below 300 mg/dL (3.42 mmol/L) or diarrhoea is mild.
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
- North Star Ambulatory Assessment [ Time Frame: 18 months ]the mean change in North Star Ambulatory Assessment
- Muscle strength evaluated by knee extension, elbow flexion [ Time Frame: 18 months ]the mean change of muscle strength evaluated by knee extension, elbow flexion as measured by hand-held myometry (HHM)
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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