A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy
- Conditions
- Duchenne Muscular DystrophyMedDRA version: 14.1Level: SOCClassification code 10028395Term: Musculoskeletal and connective tissue disordersSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2012-002566-12-IT
- Lead Sponsor
- ITALFARMACO
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 20
1. Male children aged 7 to <11 years with an immunohistochemical and molecular diagnosis of DMD. 3. Able to complete the 2 screening 6MWTs with a minimal distance of at least 250 m each. In addition, the results of these tests must be within ±30 m of each other. 4. On a stable dose of systemic corticosteroids for at least 6 months. 5. At least 6 months worth of data on the 6MWT (this will be the historical” 6MWT). From the moment of the historical 6MWT assessment(s), the child must not have received any compound that could potentially affect the 6MWT, with the exception of the stable steroid treatment.
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Initiation of systemic corticosteroid therapy within 6 months prior to the start of study drug or change in systemic corticosteroid therapy (e.g., initiation, change in type of drug, dose modification not related to body weight change, schedule modification, interruption, discontinuation, or re initiation) within 6 months prior to the start of study drug.
2. Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength since the time of the historical 6MWT and in any case within 3 months prior to the start of study treatment (e.g., growth hormone). Vitamin D, calcium, and integrators will be allowed.
3. 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To establish the histologic effects of Givinostat administered chronically at the selected daily dose;Secondary Objective: • To establish the effects of Givinostat administered chronically at the selected daily dose on functional parameters, such as the 6-Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), and performance of upper limb (PUL) • To establish the safety and tolerability of Givinostat administered chronically at the selected daily dose in children with Duchenne muscular dystrophy (DMD) • To explore the effects of Givinostat administered chronically at the selected daily dose on parameters such as magnetic resonance imaging (MRI), biomarkers, and cytokines;Primary end point(s): The primary endpoint of this study is change in the value of MFA% comparing the histology biopsies before and after 12 months of treatment with Givinostat.;Timepoint(s) of evaluation of this end point: 12 months
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Change in additional histological endpoints (i.e., cross-sectional area, inflammation, necrosis, fibrosis, and muscle regeneration) after 12 months of treatment with Givinostat at the selected daily dose • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the 6MWT • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the NSAA • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based the PUL;Timepoint(s) of evaluation of this end point: 12 months