Ignite Creation Date:
2025-12-24 @ 10:06 PM
Ignite Modification Date:
2026-01-05 @ 5:29 PM
Study NCT ID:
NCT03238235
Status:
COMPLETED
Last Update Posted:
2024-11-18
First Post:
2017-07-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
Sponsor:
Italfarmaco
Organization:
Study Overview
Official Title:
A Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD)
Status:
COMPLETED
Status Verified Date:
2024-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Objectives:
Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months.
Secondary Objectives:
* To establish the macroscopic muscle effects of Givinostat versus placebo administered over 12 months assessed by Magnetic Resonance Imaging (MRI)/Magnetic Resonance Spectroscopy (MRS).
* To determine the other histological effects of Givinostat versus placebo administered over 12 months.
* To establish the efficacy of Givinostat versus placebo administered chronically over 12 months in slowing disease progression.
* To assess the safety and tolerability of Givinostat versus placebo administered chronically.
* To evaluate the pharmacokinetic (PK) profile of Givinostat administered chronically in the target population.
* To evaluate the impact of Givinostat versus placebo administered chronically on quality of life and activities of daily living.
Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months.
Secondary Objectives:
* To establish the macroscopic muscle effects of Givinostat versus placebo administered over 12 months assessed by Magnetic Resonance Imaging (MRI)/Magnetic Resonance Spectroscopy (MRS).
* To determine the other histological effects of Givinostat versus placebo administered over 12 months.
* To establish the efficacy of Givinostat versus placebo administered chronically over 12 months in slowing disease progression.
* To assess the safety and tolerability of Givinostat versus placebo administered chronically.
* To evaluate the pharmacokinetic (PK) profile of Givinostat administered chronically in the target population.
* To evaluate the impact of Givinostat versus placebo administered chronically on quality of life and activities of daily living.
Detailed 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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2017-001629-41 | EUDRACT_NUMBER | None | View |