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: 0
• Participant gender: All

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,

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Shaare Zedek Medical Center
• Collaborator: Pfizer

Criteria

Inclusion Criteria:

• GD patients, male and female, 18 years or older

• Currently treated with enzyme replacement therapy for 5 years and more, with a stable unchanged dose in the previous 6 months

• Imaging features of significant residual bone disease defined as QCSI under 0.3 bone at risk

• Able to provide written informed consent

Exclusion Criteria:

• Currently taking another experimental drug for any condition

• Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study.

• Past exposure to Elelyso