A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD

Sponsor
Italfarmaco
Study ID
NCT01761292
Phase
PHASE1/PHASE2
Status
Completed

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Eligibility Criteria

Sex
MALE
Age
7 Years - 11 Years
Healthy Volunteers
Not accepted

Interventions

  • Givinostat — DRUG
    Givinostat, oral suspension 10 mg/mL or oral capsules 50 mg, administered orally under fed conditions at the dose of 25 mg BID, 37.5 mg BID, and 50 mg BID during Part 1 for two weeks, and 25 mg BID and 37.5 mg BID during Part 2 for 12 months. Givinostat, oral suspension 10 mg/mL, administered orally under fed conditions at the dose of 25 mg BID or 37.5 mg BID during Extension 1, and modified as per patient's weight during Extensions 2 and 3 (up to 52 months).

Study Details

The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose. The secondary objectives of Parts 1 and 2 of the study were as follows: * 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) and biomarkers * To explore the acceptability/palatability of the oral suspension * To explore whether the effects of givinostat on disease progression may be related to the type of DMD mutation. The primary objective of the Extension of the study was to evaluate the safety and tolerability of long-term administration of givinostat administered chronically at the selected daily dose in children with DMD. The secondary objectives of the Extensions were: * To establish the effects of givinostat administered chronically at the selected daily dose on muscular functional parameters, such as the 6MWT, NSAA, and PUL (Extensions 1, 2, and 3) * To explore the effects of givinostat administered chronically at the selected daily dose on parameters such as MRI (Extension 1) * To collect information related to 2 biomarkers, latent Transforming growth factor β (TGFβ) binding protein 4 (LTBP4) and osteopontin genotype (at the beginning of Extension 2 only) * To collect information related to time to wheelchair and how much time the children spend in wheelchair (Extension 3 - only for the children who were not able to complete the 6MWT)

Key Dates

Start date
Apr 30, 2013
Status verified
Jun 2020
Primary completion
Dec 31, 2014
Completion
Nov 30, 2017

Study Design

Enrollment
20 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Givinostat
    Givinostat will be administered as 2 oral doses daily while the child is in fed state.

Primary Outcome Measure

Change From Baseline to Part 2 in the Value of Muscle Fiber Area (MFA) % Comparing the Histology Biopsies Before and After 12 Months of Treatment With Givinostat. [ Time Frame: After12 months of treatment ]

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