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Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

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Disease Information

Descriptive Information
Brief Title † Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Official Title † Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Brief Summary 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: 1. Deflazacort daily regimen 2. Deflazacort intermittent regimen 3. Other steroids daily regimen 4. Other steroids intermittent regimen. The study duration is planned for 19 months.
Detailed Description 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 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 Phase Phase 3
Study Type † Interventional
Study Design †
Primary Outcome Measure † mean change in 4 standard stairs climb
Secondary Outcome Measure † Other functional test as 6MWT
Condition † Duchenne Muscular Dystrophy
Intervention † Druggivinostat
Study Arms / Comparison Groups givinostat Givinostat oral suspension (10 mg/mL) twice daily in a fed state placebo Placebo oral suspension (10 mg/mL) twice daily in a fed state
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 213
Start Date † April 2017
Completion Date June 2020
Primary Completion Date June 2020
Eligibility Criteria † Inclusion Criteria: 1. Are an ambulant male aged ≥6 years at randomisation with DMD characteristic clinical symptoms or signs (e.g., proximal muscle weakness, Gowers' maneuver, elevated serum creatinine kinase level) already present at screening; 2. Have DMD diagnosis confirmed by genetic testing; 3. Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations); 4. Are able to complete 2 Four Stairs Climb test (4SC) screening assessments; the results of these tests must be within ±1 second of each other; 5. Have the mean of 2 screening 4SC assessments ≤8 seconds; 6. Have time to rise from floor of 10% but 2 x the upper limit of normal (ULN); 14. Have a positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening; 15. Have a baseline QTcF >450 msec, or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome); 16. Have a psychiatric illness/social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures; 17. Have any known allergic reaction to givinostat or any of its excipients. For the subgroup of subjects who will undergo MRI and MRS (i.e., MR Cohort): 18. Have contraindications to MRI or MRS (e.g., claustrophobia, metal implants, or seizure disorder).
Gender Male
Ages 6 Years - 17 Years
Accepts Healthy Volunteers No
Contacts †† Paolo Bettica, MD, +39 0264432511, p.bettica@italfarmaco.com
Location Countries † Belgium
Administrative Information
NCT ID † NCT02851797
Organization ID DSC/14/2357/48
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Italfarmaco
Collaborators †† inVentiv Health Clinical
Investigators † : ,
Information Provided By
Verification Date March 2017
First Received Date † July 27, 2016
Last Updated Date March 29, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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