Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
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ClinicalTrials.gov Identifier: NCT03373968 |
Recruitment Status :
Enrolling by invitation
First Posted : December 14, 2017
Last Update Posted : November 7, 2023
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Tracking Information | |||||
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First Submitted Date ICMJE | December 4, 2017 | ||||
First Posted Date ICMJE | December 14, 2017 | ||||
Last Update Posted Date | November 7, 2023 | ||||
Actual Study Start Date ICMJE | October 24, 2017 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through study completion, an average of 1 year ] Type, incidence, and severity of treatment related/not related adverse events(AEs) and serious adverse event (SAEs)
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study | ||||
Official Title ICMJE | Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies | ||||
Brief Summary | This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies. | ||||
Detailed Description | GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term study will be the same that the subject was receiving at the end of the previous DMD GIVINOSTAT study. As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according the rules detailed in the study protocol section 11.2.2.1. 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 meet the stopping criteria for platelets, the Investigator will have to reduce the dose of 1/3 or 20% less of the current dose as described in the study protocol section 10.5.1.3. During the first month of treatment, platelets count assessment will be performed weekly, while during the second month it will be performed every 2 weeks, in order to strictly monitor this parameter for safety reasons, with the exclusion of subjects coming from study DSC/11/2357/43 for which the first visit will be 4 months after the Visit 1/baseline visit. Study drug should be permanently interrupted if any of the following occurs:
The Investigator will follow up the patient until resolution or acceptable stabilization of the event occurs and document all the relevant information, as applicable. After the resolution/stabilization of the event, the subject will be withdrawn from the study and the EOS Visit (see Section 12.1.10) will be performed. Any decision relevant to the dose adjustment and/or modification of schedule of assessments can be discussed with the Medical Monitor, but the final decision remains with the Investigator only or its authorized designee. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 2 Phase 3 |
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Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE | Drug: Givinostat
suspension of givinostat (10 mg/mL)
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Study Arms ICMJE | Experimental: givinostat
Givinostat oral suspension (10 mg/mL) twice daily in a fed state
Intervention: Drug: Givinostat
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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 | Enrolling by invitation | ||||
Estimated Enrollment ICMJE |
206 | ||||
Original Estimated Enrollment ICMJE |
100 | ||||
Estimated Study Completion Date ICMJE | December 2025 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 7 Years and older (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Serbia, Spain, United Kingdom, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03373968 | ||||
Other Study ID Numbers ICMJE | DSC/14/2357/51 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Italfarmaco | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Italfarmaco | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Cromsource | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | Italfarmaco | ||||
Verification Date | November 2023 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |