Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
NCT04353466
ABOUT THIS STUDY
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- 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
- 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
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Descriptive Information | |||||||
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Brief Title ICMJE | Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs | ||||||
Official Title ICMJE | 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 | ||||||
Brief 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 | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Not Applicable | ||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: The objective of this study is to assess Elelyso treatment on bone disease in Gaucher patients currently treated with other enzyme replacement therapy Masking: None (Open Label)Primary Purpose: Treatment | ||||||
Condition ICMJE | Gaucher Disease, Type 1 | ||||||
Intervention ICMJE |
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Study Arms ICMJE | Experimental: Trial to asses impact of Elelyso on bone involvement in patien
The infusions will be administered at the selected medical center or in the home care setup. The dose of intravenous (IV) infusions 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. Interventions:
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||||
Recruitment Information | |||||||
Recruitment Status ICMJE | Active, not recruiting | ||||||
Actual Enrollment ICMJE | 30 | ||||||
Original Actual Enrollment ICMJE | Same as current | ||||||
Estimated Study Completion Date ICMJE | July 31, 2021 | ||||||
Estimated Primary Completion Date | July 31, 2021 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | Not Provided | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT04353466 | ||||||
Other Study ID Numbers ICMJE | 138-16-SZMC | ||||||
Has Data Monitoring Committee | No | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Ari Zimran, Shaare Zedek Medical Center | ||||||
Study Sponsor ICMJE | Shaare Zedek Medical Center | ||||||
Collaborators ICMJE | Pfizer | ||||||
Investigators ICMJE |
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PRS Account | Shaare Zedek Medical Center | ||||||
Verification Date | April 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |