Study in which all the patients take the same investigational drug with theaim of evaluate the long-term safety, tolerability, and efficacy ofGIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophywho have been already treated in one of the GIVINOSTAT studies in thepast

Phase 1

• Conditions: Duchenne Muscular Dystrophy (DMD); MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2017-000397-10-GB
• Lead Sponsor: ITALFARMACO S.p.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-03-15
• Last Update Posted: 9 September 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 185

Inclusion Criteria

To clarify A.15 Answer:
Male subjects affected by Duchenne Muscular Dystrophy previously enrolled in one of the other DMD GIVINOSTAT studies (e.g DSC/14/2357/43, DSC/14/2357/48 and DSC/14/2357/50), aged =7 years and 6 months

INCLUSION CRITERIA:
1. Must have participated in one of the previous studies with
GIVINOSTAT in DMD and have attended the End of Study Visit;
2.Aged =7 years and 6 months 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 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.
oCondom 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.

Are the trial subjects under 18? yes
Number of subjects for this age range: 175
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

Patients who meet any of the following criteria will NOT be eligible for enrolment into the trial:

Subjects presenting with any of the following criteria will not be
included in the study:
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 (be repeated once; if the repeat test result is still exclusionary);
6.Have Triglycerides > 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 clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the long term safety and tolerability of GIVINOSTAT oral suspension in patients with Duchenne Muscular Dystrophy (DMD) following core protocols program.;Secondary Objective: To evaluate the effects of long-term administration of GIVINOSTAT on muscular function and strength; <br>To evaluate the effects of long-term administration of GIVINOSTAT on respiratory function;<br>To evaluate the impact on daily activities and quality of life following long-term administration of GIVINOSTAT.<br>;Primary end point(s): Type, incidence, and severity of treatment related/not related AEs and<br>SAEs<br><br>;Timepoint(s) of evaluation of this end point: Baseline through week 48 and then yearly till the end of the study<br>