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 : February 10, 2020
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 10^9/L.
- white blood cells ≤2.0 x 10^9/L
- hemoglobin ≤8.0 g/dL
Study drug should be temporarily stopped if any of the following occur:
- moderate or severe diarrhoea.
- platelets count <75 x 10^9/L but >50 x 10^9/L (the treatment should be temporarily stopped and a platelets count has to be performed and re-tested until platelets will be normalized);
- white blood cell <3.0 x 10^9/L but >2.0 x 10^9/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 20% 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.
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 meeting the stopping criteria for platelets, the Investigator will have to reduce the dose by 20% of the current dose.
Only one dose reduction is allowed during the treatment period.
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 :||March 2022|
|Estimated Study Completion Date :||March 2022|
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)
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|