Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
Status:
Active, not recruiting
Trial end date:
2021-07-31
Target enrollment:
Participant gender:
Summary
The objective of this study is to assess Elelyso treatment on bone disease in Gaucher
patients currently treated with other enzyme replacement therapy.
Experience from early access program (2009-2012) has suggested that some patients who have
been stable on imiglucerase have shown poor scores of QCSI with Fat Fraction below the cut
off point of 0.23 which is considered "bone at risk", and have demonstrated remarkable
improvement upon switching to Elelyso, including particularly 2 patients who did not have any
change in dose or any drug interruption prior to the switch.
These findings may be explained by the better glycan structure of imiglucerase (see Tekoah et
al, 2013). The fact that in many patients prevention of bony complications is the main
indication for ERT highlights the importance of this study, as all clinical trials of all
ERTS heretofore did not include the bones as primary or secondary end-points but only as
exploratory, and as such had only limited value,