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

NCT ID: NCT04353466

Last Updated: 2022-10-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-01
• Study Completion Date: 2021-07-31

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

Conditions

Gaucher Disease, Type 1

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

quantitative chemical shift imaging (QCSI)

Intervention Type: PROCEDURE

Poor scores of QCSI with Fat Fraction below the cut off point of 0.3 which is considered "bone at risk"

Elelyso

Intervention Type: DRUG

intravenous (IV) infusions of Elelyso

Interventions

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"

Intervention Type: PROCEDURE

Elelyso

intravenous (IV) infusions of Elelyso

Intervention Type: DRUG

Other Intervention Names

Taliglucerase Alfa

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: collaborator

Shaare Zedek Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Ari Zimran

Prof.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Ari Zimran, MD

Role: PRINCIPAL_INVESTIGATOR

Shaheed Ziaur Rahman Medical College

References

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 Mol Dis. 2013 Mar;50(3):206-11. doi: 10.1016/j.bcmd.2012.11.001. Epub 2012 Nov 28.

Reference Type: RESULT

PMID: 23199589 (View on PubMed)

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 Nov;41(6):1259-1265. doi: 10.1007/s10545-018-0195-y. Epub 2018 Jul 31.

Reference Type: RESULT

PMID: 30066229 (View on PubMed)

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. Epub 2014 Jun 13.

Reference Type: RESULT

PMID: 24924293 (View on PubMed)

Other Identifiers

138-16-SZMC

Identifier Type: -

Identifier Source: org_study_id