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completed

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

key information

study id #: NCT01761292

condition: Duchenne Muscular Dystrophy (DMD)

status: completed

purpose:

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)

 

intervention: Givinostat

mechanism of action: Histone deacetylase (HDAC) inhibitor to reduce inflammation and promote muscle health

results: https://clinicaltrials.gov/ct2/show/results/NCT01761292

last updated: July 06, 2020

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