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 :
Active, not recruiting
First Posted : August 2, 2016
Last Update Posted : September 21, 2020
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Tracking Information | |||||
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First Submitted Date ICMJE | July 27, 2016 | ||||
First Posted Date ICMJE | August 2, 2016 | ||||
Last Update Posted Date | September 21, 2020 | ||||
Actual Study Start Date ICMJE | June 1, 2017 | ||||
Estimated Primary Completion Date | March 2022 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy | ||||
Official Title ICMJE | 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:
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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:
Study drug should be temporarily stopped if any of the following occur:
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. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 3 | ||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Estimated Enrollment ICMJE |
169 | ||||
Original Estimated Enrollment ICMJE |
213 | ||||
Estimated Study Completion Date ICMJE | March 2022 | ||||
Estimated Primary Completion Date | March 2022 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
At the discretion of the Investigator, subjects not meeting inclusion/exclusion criteria may be re-screened twice with an interval of at least 3 months between assessments. |
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Years to 17 Years (Child) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Netherlands, Belgium, Canada, France, Germany, Israel, Italy, Serbia, Spain, United Kingdom, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT02851797 | ||||
Other Study ID Numbers ICMJE | EPYDIS (DSC/14/2357/48) | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Italfarmaco | ||||
Study Sponsor ICMJE | Italfarmaco | ||||
Collaborators ICMJE | Syneos Health | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | Italfarmaco | ||||
Verification Date | September 2020 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |