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

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Brief Title

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

Official Title

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

Brief Summary

      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.
    

Detailed Description

      GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily
      while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term
      study will be the same that the subject was receiving at the end of the previous DMD
      GIVINOSTAT study.

      As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight
      according the rules detailed in the study protocol section 11.2.2.1.

      In addition, in case a subject will have a consistent (e.g., at least 2 consecutive
      evaluations) platelets count ≤150 x 10^9/L and not meet the stopping criteria for platelets,
      the Investigator will have to reduce the dose of 1/3 or 20% less of the current dose as
      described in the study protocol section 10.5.1.3. During the first month of treatment,
      platelets count assessment will be performed weekly, while during the second month it will be
      performed every 2 weeks, in order to strictly monitor this parameter for safety reasons, with
      the exclusion of subjects coming from study DSC/11/2357/43 for which the first visit will be
      4 months after the Visit 1/baseline visit.

      Study drug should be permanently interrupted if any of the following occurs:

        -  severe drug-related diarrhoea (i.e., increase of ≥7 stools per day);

        -  any drug-related SAE;

        -  QTcF >500 msec;

        -  platelets count ≤50 x 10^9/L;

        -  white blood cells ≤2.0 x 10^9/L;

        -  hemoglobin ≤8.0 g/dL; To avoid laboratory errors and anomalous values, test must be
           confirmed with a repeated test performed on the next working day. The treatment should
           be stopped until the retest result becomes available. If the repeated test is still
           under the stopping limit value, study drug must be permanently discontinued. If the
           repeated test is acceptable, the subject can resume treatment.

      The Investigator will follow up the patient until resolution or acceptable stabilization of
      the event occurs and document all the relevant information, as applicable. After the
      resolution/stabilization of the event, the subject will be withdrawn from the study and the
      EOS Visit (see Section 12.1.10) will be performed.

      Any decision relevant to the dose adjustment and/or modification of schedule of assessments
      can be discussed with the Medical Monitor, but the final decision remains with the
      Investigator only or its authorized designee.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]


Condition

Duchenne Muscular Dystrophy

Intervention

Givinostat

Study Arms / Comparison Groups

 givinostat
Description:  Givinostat oral suspension (10 mg/mL) twice daily in a fed state

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

206

Start Date

October 24, 2017

Completion Date

December 2023

Primary Completion Date

December 2023

Eligibility Criteria

        Inclusion Criteria:

          1. Must have participated in one of the previous studies 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:

               -  all the inclusion criteria and none of the exclusion criteria,

               -  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,

               -  never been randomized because, the enrollment 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 localregulations);

          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.

                    -  Condom with spermicide and the female partner must use an acceptable method
                       of contraception, such as an oral,

                    -  transdermal, injectable or implanted steroid-basedcontraceptive, or a
                       diaphragm or a barrier method of contraception in conjunction with
                       spermicidal jelly such asfor example cervical cap with spermicide jelly.

        Exclusion Criteria:

          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 (>300 mg/dL), the triglycerides will
             be repeated once; if the repeat test result is still >300 mg/dL, 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 situation 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.

               -  the Investigators to evaluate these exclusion criteria can use the laboratory
                  results obtained within 5 months from V1, to allow the continuity of the
                  treatment. It is worth noting, as soon as the site will receive the laboratory
                  results done in screening/baseline (Visit 1) visit they will check the GIVINOSTAT
                  dose and modify it as per protocol safety rules and/or dosage modifications
                  rules.
      

Gender

Male

Ages

7 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Belgium

Location Countries

Belgium

Administrative Informations


NCT ID

NCT03373968

Organization ID

DSC/14/2357/51


Responsible Party

Sponsor

Study Sponsor

Italfarmaco

Collaborators

 Cromsource

Study Sponsor

, , 


Verification Date

March 2022