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About
Objectives:
Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months.
Secondary Objectives:
Full description
This was a phase 2, randomised, double-blind, placebo-controlled study. Eligible patients were randomized in a 2:1 ratio to receive Givinostat or placebo for 12 months. Randomization was stratified by concomitant steroid use at baseline (yes or no). The study comprised twelve (12) visits: screening (V1, V2), randomization (V3), treatment (V4-V10), end of study (V11) and follow-up (V12). Visits during treatment took place every 12 weeks, except for the first 2 months, when they occurred every 2 weeks to allow closer monitoring of safety.
Givinostat (ITF2357) oral suspension (10 mg/mL) was initially administered as 2 daily doses of 40-70 mg according to body weight after a meal (high dose). With amendment 2 of the protocol, a lower starting dose was implemented to address cases of thrombocytopenia reported following the treatment of the first 21 patients and corresponded to the reduced dose of the original protocol (i.e., 26.7-46.7 mg b.i.d according to body weight, i.e., low dose).
51 patients were to be enrolled to provide a sample size of 48 patients with evaluable baseline biopsies. Seventy patients provided written informed consent, 51 (72.86%) completed screening successfully and were randomized; 34 patients (66.67%) to the Givinostat group and 17 (33.33%) to the placebo group.
Enrollment
Sex
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Volunteers
Inclusion criteria
Ambulant patients with BMD diagnosis confirmed by genetic testing.
Able and willing to give informed consent in writing.
Able to perform 6MWT at screening with a minimum distance of 200 m and maximum distance of 450 m.
If in treatment with systemic corticosteroids and/or angiotensin-converting-enzyme (ACE) inhibitor , and/or β or α adrenergic receptor blocker, no significant change in dosage or dosing regimen (excluding changes related to body weight) was to be presented for a minimum of 6 months prior to start of study treatment.
Patients had to be willing to use adequate contraception from randomization until 3 months after the last dose of study treatment, and included the following:
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
51 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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