welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability Study
study id #: NCT03373968
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: Histone deacetylase (HDAC) inhibitor to reduce inflammation and promote muscle health
last updated: November 22, 2018
start date: October 24, 2017
estimated completion: December 2023
phase of development: Phase 2/Phase 3
size / enrollment: 100
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
- 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)
• 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.
• 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.
Phase 1/2 Study in Boys With Duchenne Muscular DystrophyThe MoveDMD study is a 3-part, Phase 1/2...
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyThe primary objective of this study is t...
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double...Background: Duchenne muscular dystrophy...
PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two ...PTC Therapeutics, Inc. today announced t...
Design of a phase 3 trial to evaluate the long-term efficacy and safety of ataluren in patients with nonsense mutati...Ataluren is conditionally approved by th...
PTC Therapeutics Phase III Duchenne Muscular Dystrophy (DMD) Clinical TrialPTC Therapeutics, Inc. is seeking pati...
Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study ReportsVamorolone, a Duchenne muscular dystroph...