Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS

ITALFARMACO S.p.A.0 sites169 target enrollmentOctober 26, 2016

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Not specified
Sponsor: ITALFARMACO S.p.A.
Enrollment: 169
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

October 26, 2016

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Investigators

Sponsor

ITALFARMACO S.p.A.

Eligibility Criteria

Inclusion Criteria

•1\. Are ambulant males aged \=6 years at randomisation with DMD characteristic clinical symptoms or signs (e.g., proximal muscle weakness, Gowers’ maneuver, elevated serum creatinine kinase level) already present at screening;
•2\. Have DMD diagnosis confirmed by genetic testing;
•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\. Are able to complete 2 Four Stairs Climb test (4SC) screening assessments; the results of these tests must be within ±1 second of each other;
•5\. Have the mean of 2 screening 4SC assessments \=8 seconds;
•6\. Have time to rise from floor between \=3 and \<10 seconds at screening;
•7\. Have manual muscle testing (MMT) of quadriceps at screening \= Grade \- 3;
•8\. Have used systemic corticosteroids for a minimum of 6 months immediately prior to the start of study treatment, with no significant change in corticosteroids type or dosage or dosing regimen (excluding changes related to body weight change) for a minimum of 6 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
•9\. Subjects must be willing to use adequate contraception.
•Contraceptive methods must be used from Randomization Visit 3 through 3 months after the last dose of study drug, and include the following:

Exclusion Criteria

•1\. Have exposure to another investigational drug within 3 months prior to the start of study treatment (only exception allowed is use of Deflazacort in US as part of the Expanded Access Program and in Canada as part of the Special Access Program);
•2\. Have exposure to idebenone within 3 months prior to the start of study treatment;
•3\. Have exposure to any dystrophin restoration product (e.g., Ataluren, Exon\-skipping) within 6 months prior to the start of study treatment;
•4\. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed as long as their intake has been stable for 3 months prior to the start of study treatment. Testosterone will also be allowed if it is used as a replacement therapy for the treatment of delayed puberty, and testosterone dose and regimen have been stable for at least 6 months and circulating testosterone levels are within the normal ranges for the subject’s age;
•5\. Have surgery that might have an effect on muscle strength or function within 3 months before study entry or planned surgery at any time during the study;
•6\. Loss of \=30 degrees of plantar flexion from the normal range of movement at the ankle joint due to contracture (i.e. fixed loss of more than 10 degrees of plantar flexion from plantigrade, assuming normal range of dorsiflexion of 20 degrees) ;
•7\. Change in contracture treatment such as serial casting, contracture control devices, night splints, stretching exercises (passive, active, self) within 3 months prior to enrollment, or expected need for such intervention during the study;
•8\. Have presence of other clinically significant disease, which, in the Investigator’s opinion, could adversely affect the safetyof the subject, making it unlikely that the course of treatment or follow\-up would be completed, or could impair the assessment of study results;
•9\. Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;
•10\. Have platelets count, White Blood Cell and Hemoglobin at screening 11\. Have symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) or left ventricular ejection fraction \<50% at screening;