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

NCT04353466

Last updated date
Contact
1-800-718-1021

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting the study website. Please see the references below:

By phone

Pfizer Clinical Trials Contact Center

1-800-718-1021

By email

Contact

[email protected]

Call Now

Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Type 1 Gaucher Disease
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


- 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

NEED INFO?

Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

Pfizer Clinical Trials Contact Center

1-800-718-1021

[email protected]

TRY A NEW SEARCH

Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.

Based on your search, you may also be interested in

Type 1 Gaucher DiseaseAssessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
NCT04353466
ALL GENDERS
18 Years+
years
MULTIPLE SITES
Type 1 Gaucher DiseasePharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
NCT03021941
  1. Fairfax, Virginia
ALL GENDERS
0+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
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
  • 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
    Other Name: Taliglucerase Alfa
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:
  • Procedure: quantitative chemical shift imaging (QCSI)
  • Drug: Elelyso
Publications *

*   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
 (submitted: April 16, 2020)
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:

  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study:All
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
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:No
Responsible Party Ari Zimran, Shaare Zedek Medical Center
Study Sponsor  ICMJE Shaare Zedek Medical Center
Collaborators  ICMJE Pfizer
Investigators  ICMJE
Principal Investigator:Ari Zimran, MDShaheed Ziaur Rahman Medical College
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