NCT04353466

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

An Open-label, Investigator Initiated Clinical Trial to Asses Impact of Elelyso on Bone Involvement in Patients With Gaucher Disease Currently Treated With Other ERTs

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Shaare Zedek Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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,

Detailed description

Open-label study in patients with Gaucher disease currently treated with commercial ERTs. Eligible patients will receive intravenous (IV) infusions of Elelyso every two weeks. The infusions will be administered at the selected medical center or in the home care setup. The dose of Elelyso will be the same dose of the other ERTs . Bone parameters QCSI and BMD will be assessed at baseline, 12 months and 24 months. The intention is to open 3 more sites in Israel thereby making this IIR a multi center national trial

Conditions

• Gaucher Disease, Type 1

Interventions

Type	Name	Description
PROCEDURE	quantitative chemical shift imaging (QCSI)	Poor scores of QCSI with Fat Fraction below the cut off point of 0.3 which is considered "bone at risk"
DRUG	Elelyso	intravenous (IV) infusions of Elelyso

Timeline

• Start date: 2017-01-01
• Primary completion: 2021-07-31
• Completion: 2021-07-31
• First posted: 2020-04-20
• Last updated: 2022-10-28