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A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

NCT04002830 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2024-12-17

Study results available
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Summary

This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.

Conditions

  • Gaucher Disease, Type 3

Interventions

DRUG

Elelyso

Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.

Sponsors & Collaborators

  • Pfizer

    collaborator INDUSTRY

  • Ari Zimran

    lead OTHER

Principal Investigators

  • Ari Zimran, Prof. · Shaare Zedek Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-20
Primary Completion
2023-07-30
Completion
2023-07-30
FDA Drug
Yes

Countries

  • India
  • Israel
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Companies

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 NCT04002830 on ClinicalTrials.gov