A Long-Term Safety and Tolerability Study of Experimental Givinostat in Duchenne Muscular Dystrophy

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Age: 7 years or older
Gender:
Male
Healthy Volunteers: No
Keywords: DMD, duchenne muscular dystrophy
Type: Drug study, Phase [2, 3]
Target:
5 Participants
Investigator:
Description
This study will test the long-term safety and effectiveness of the experimental medicine Givinostat. It will be given to patients with Duchenne Muscular Dystrophy (DMD).

This long-term study is for patients who have already participated in a study with Givinostat. It provides the opportunity for them to continue treatment.

This study is for patients with Duchenne Muscular Dystrophy currently taking experimental Givinostat.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  • Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit;
  • Aged ≥7 years and 6 months old
  • 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 uncontrolled neurological diseases or presence of relevant uncontrolled 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 Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting conditionat screening visit (for abnormal screening laboratory test results (>300 mg/dL), the triglycerides will be repeated once;if the repeat test result is still >300 mg/dL , then exclusionary).
  • Have inadequate renal function, as defined by serum Cystatin C >2 x the upper limit of normal (ULN) at screening visit. If the value is >2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 x ULN, the subject should be excluded);
  • 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.
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
Your participation in this research will last approximately 7 years. The study visits for this study will occur every 4 months through the entire study. If you are currently receiving treatment in a study that also involves a placebo, you will need to: • First month – attend weekly visits • Second month – attend visits every two weeks and then visits at the end of the third and fourth months. • From month four, visits will be every four months until the end of the study
Recruitment period
From June 19, 2019
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Laura Pulido-Ramirez
Research Topic
Conditions:
  • Duchenne Muscular Dystrophy