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

ITALFARMACO S.p.A.0 sites192 target enrollmentFebruary 8, 2017

ConditionsDuchenne Muscular Dystrophy (DMD)MedDRA version: 19.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Duchenne Muscular Dystrophy (DMD)
Sponsor: ITALFARMACO S.p.A.
Enrollment: 192
Status: Active, not recruiting
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

February 8, 2017

End Date

TBD

Last Updated

8 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 of \<10 seconds at screening;
•7\. Have manual muscle testing (MMT) of quadriceps at screening \= Grade 3;
•8\. Are eligible according to the protocol\-specified functional algorithm predictive of vastus lateralis muscle fat fraction (VL MFF) that should be in the range \>10% but \<30%;
•9\. Have used systemic corticosteroids for a minimum of 6 months immediately prior to the start of study treatment, with no significant change in 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.
•10\. Subjects must be willing to use adequate contraception.

Exclusion Criteria

•1\. Have exposure to another investigational drug within 3 months prior to the start of study treatment;
•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;
•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\. Ankle joint contractures due to a fixed loss of \=10 degrees of dorsiflexion from plantagrade assuming a normal range of dorsiflexion of 20 degree;
•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 neurological diseases or presence of relevant 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;