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

NCT04002830 | Completed Phase 4 Results DMC FDA Drug

• 1 other identifier: WI224302
• Study Type: interventional
• Target: 14
• Locations: 3 countries
• Sites: 3

Brief 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

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in Spleen Volume Measured by MRI

  Percent change from baseline

  from baseline to month 12

Secondary Outcomes (4)

• Percent Change From Baseline in Liver Volume Measured by MRI

  from baseline to month 12

• Percent Change in Hemoglobin

  from baseline to Months 3, 6, 9, and 12

• Percent Change in Platelet Count

  from baseline to Months 3, 6, 9, and 12

• Percent Change in Lyso-GB1

  from baseline to Months 3, 6, 9, and 12

Study Arms (1)

Taliglucerase Alpha

EXPERIMENTAL

Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease

Drug: Elelyso

Interventions

Elelyso DRUG

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.

Also known as: Taliglucerase Alfa

Taliglucerase Alpha

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female of any age; however, if female:
• must be using contraception if of childbearing potential or must be surgically sterile
• must not be lactating
• Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor.
• Splenomegaly at least 5 x multiples of normal (MN).
• Treatment-naïve.

You may not qualify if:

• Type 2 GD.
• Presence of myoclonic seizures.
• At least one allele of:
• N370S (N409S in recent nomenclature)
• R496H (R535H in recent nomenclature)
• Presence of calcification in heart valves or arteries in echocardiography.
• Presence of human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and/or hepatitis C infections.
• Splenectomy and bone marrow transplantation.
• Presence of any medical, emotional, behavioural, or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
• Any other disorder that may interfere with the results of the efficacy endpoints.
• Pregnancy or breastfeeding.
• Currently taking another investigational drug for any condition or any therapeutic drug for Gaucher disease.
• The subject and/or subject's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study.
• Medical history of any food/drugs allergy.

Sponsors & Collaborators

• Ari Zimran: lead
• Pfizer: collaborator

Study Sites (3)

All India Institute of Medical Sciences

New Delhi, India

Location

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

Location

Gazi University

Ankara, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

taliglucerase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Majdolen Istaiti
• Organization: Shaare Zedek Medical Center

joleenist@szmc.org.il

972526659995

Study Officials

• Ari Zimran, Prof.

  Shaare Zedek Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: PI

Model Details: Type 3 Gaucher disease patients

Study Record Dates

• First Submitted: June 27, 2019
• First Posted: July 1, 2019
• Study Start: November 20, 2020
• Primary Completion: July 30, 2023
• Study Completion: July 30, 2023
• Last Updated: December 17, 2024
• Results First Posted: December 17, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

IN (1); IL (1); TU (1)