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.
- Conditions
- Congenitalhereditary muscular dystrophy10028396
- Registration Number
- NL-OMON54775
- Lead Sponsor
- CROMSOURCE BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 16
Subjects must meet all of the following criteria in order to be included in the
study: 1. Must have participate in one of the previous study with GIVINOSTAT in
DMD and have attended the End of Study Visit or must have been screened in
study DSC/14/2357/48 and met: o all the inclusion criteria and none of the
exclusion criteria, o had a baseline vastus lateralis muscle fat fraction (VL
MFF) assessed by MRS in the range <=5% or >30%, i.e. included in *off-target*
group, o never been randomized because, the enrolment in the off-target group
was completed. 2. Aged >=6 years old 3. Are able to give informed assent and/or
consent in writing signed by the subject and/or parent/legal guardian
(according to local regulations); 4. Subjects must be willing to use adequate
contraception: Contraceptive methods must be used since the previous GIVINOSTAT
study through 3 months after the last dose of study drug, and include the
following: * True abstinence (absence of any sexual intercourse), when in line
with the preferred and usual lifestyle of the subject. Periodic abstinence
(e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal
are not acceptable methods of contraception. * Condom with spermicide and the
female partner must use an acceptable method of contraception, such as an oral,
transdermal, injectable or implanted steroid-based contraceptive, or a
diaphragm or a barrier method of contraception in conjunction with spermicidal
jelly such as for example cervical cap with spermicide jelly. GIVINO
Patients who meet any of the following criteria will NOT be eligible for
enrolment into the trial: 1. 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; 2. Use of any
current investigational drug other than Givinostat; 3. 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; 4. Have a diagnosis of other uncontrolled neurological diseases
or presence of relevant uncontrolled somatic disorders that are not related to
DMD; 5. Have platelets count, White Blood Cell and Hemoglobin at screening <
Lower Limit of Normal (LLN)* (for abnormal screening laboratory test results (
300 mg/dL (3.42 mmol/L) in fasting condition at screening visit* (for abnormal
screening laboratory test results (>ULN), the triglycerides will be repeated
once; if the repeat test result is still >ULN, then exclusionary), 7. 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) 8. Have heart failure (New York
Heart Association Class III or IV); 9. 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; 10. Have a baseline 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); 11. 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. 12. Have any hypersensitivity
to the components of study medication; 13. Have a sorbitol intolerance or
sorbitol malabsorption, or have the hereditary form of fructose intolerance.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Type, incidence, and severity of treatment related/not related AEs and SAEs<br /><br>(Baseline through week 48 and then yearly till the end of the study)</p><br>
- Secondary Outcome Measures
Name Time Method