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

NCT04353466 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2022-10-28

No results posted yet for this study

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,

Conditions

  • Gaucher Disease, Type 1

Interventions

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

Sponsors & Collaborators

  • Pfizer

    collaborator INDUSTRY

  • Shaare Zedek Medical Center

    lead OTHER

Principal Investigators

  • Ari Zimran, MD · Shaheed Ziaur Rahman Medical College

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-01-01
Primary Completion
2021-07-31
Completion
2021-07-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04353466 on ClinicalTrials.gov