A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
Status:
Completed
Trial end date:
2017-11-01
Target enrollment:
Participant gender:
Summary
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)