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Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

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ClinicalTrials.gov Identifier: NCT03373968
Recruitment Status : Recruiting
First Posted : December 14, 2017
Last Update Posted : December 18, 2017
Sponsor:
Collaborator:
Cromsource
Information provided by (Responsible Party):
Italfarmaco

December 4, 2017
December 14, 2017
December 18, 2017
October 24, 2017
December 2023   (Final data collection date for primary outcome measure)
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)
Same as current
Complete list of historical versions of study NCT03373968 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
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
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.

Givinostat oral suspension (10 mg/mL) will be administered orally as 2 oral doses daily while the subject is in fed state. The subjects will continue to receive the same dose that has been taken in the previous study. Moreover, as givinostat exposures are influenced by subjects'weight, the dose will be adjusted by the subjects weight gain during the trial.

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 10e9/L.

Study drug should be temporarily stopped if any of the following occur:

  • platelets count <75 x 10e9/L but >50 x 10e9/L (the treatment should be temporarily stopped and a platelets count has to be performed and retested 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).

The first interim analysis will be performed after 48 weeks after the First Subject First Visit and then yearly till the marketing authorization will be granted. In each Interim Analysis all safety and efficacy variables will be analyzed.

Interventional
Phase 2
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Drug: Givinostat
suspension of givinostat (10 mg/mL)
Experimental: givinostat
Givinostat oral suspension (10 mg/mL) twice daily in a fed state
Intervention: Drug: Givinostat
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
100
December 2023
December 2023   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit;
  • Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations);
  • Subjects must be willing to use adequate contraception.

Exclusion Criteria:

  • Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;
  • Use of any current investigational drug other than Givinostat;
  • Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;
  • Have a diagnosis of other neurological diseases or presence of relevant somatic disorders that are not related to DMD;
  • Have platelets count, White Blood Cell and Hemoglobin at screening < Lower Limit of Normal (LLN) (for abnormal screening laboratory test results (<LLN), the platelets count, White Blood Cell and Hemoglobin will be repeated once; if the repeat test result is still <LLN, then exclusionary);
  • Have heart failure (New York Heart Association Class III or IV)
  • Have a current liver disease or impairment, including but not limited to an elevated total bilirubin (i.e. > 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;
  • Have a baseline QT interval, Fredericia's correction, (QTcF) >450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);
  • 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.
  • Have any hypersensitivity to the components of study medication;
  • Have a sorbitol intolerance or sorbitol malabsorption, or have the hereditary form of fructose intolerance.
Sexes Eligible for Study: Male
7 Years and older   (Child, Adult, Senior)
No
Contact: Paolo Bettica, MD PhD + 39 02 6443 ext 2511 p.bettica@italfarmaco.com
Contact: Sara Cazzaniga, Bio. Sc. + 39 02 6443 ext 2524 s.cazzaniga@italfarmaco.com
Italy
 
 
NCT03373968
DSC/14/2357/51
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: Undecided
Italfarmaco
Italfarmaco
Cromsource
Not Provided
Italfarmaco
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP