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

Gaucher Disease, Type 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04353466
• Other study ID #: 138-16-SZMC
• Status: Completed
• Phase: N/A
• Start date: January 1, 2017
• Est. completion date: July 31, 2021

Study information

• Verified date: October 2022
• Source: Shaare Zedek Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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,

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: July 31, 2021
• Est. primary completion date: July 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type 1

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Shaare Zedek Medical Center	Pfizer

References & Publications (3)

van Dussen L, Akkerman EM, Hollak CE, Nederveen AJ, Maas M. Evaluation of an imaging biomarker, Dixon quantitative chemical shift imaging, in Gaucher disease: lessons learned. J Inherit Metab Dis. 2014 Nov;37(6):1003-11. doi: 10.1007/s10545-014-9726-3. Ep — View Citation

van Dussen L, Zimran A, Akkerman EM, Aerts JM, Petakov M, Elstein D, Rosenbaum H, Aviezer D, Brill-Almon E, Chertkoff R, Maas M, Hollak CE. Taliglucerase alfa leads to favorable bone marrow responses in patients with type I Gaucher disease. Blood Cells Mo — View Citation

Zimran A, Dinur T, Revel-Vilk S, Akkerman EM, van Dussen L, Hollak CEM, Maayan H, Altarescu G, Chertkoff R, Maas M. Improvement in bone marrow infiltration in patients with type I Gaucher disease treated with taliglucerase alfa. J Inherit Metab Dis. 2018 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	QCSI results at 12 months, Fat fraction under 0.3.	Improvement of QCSI results 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 12 months.	12 months
Primary	QCSI results at 24 months, Fat fraction under 0.3.	Improvement of QCSI results 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 24 months.	24 months