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

Primary Purpose

Gaucher Disease, Type 3

Status
Active
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Elelyso
Sponsored by
Ari Zimran
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease, Type 3

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. 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
  2. Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor.
  3. Splenomegaly at least 5 x multiples of normal (MN).
  4. Treatment-naïve.

Exclusion Criteria:

Eligible subjects may not have any of the following exclusion criteria:

  1. Type 2 GD.
  2. Presence of myoclonic seizures.

  3. At least one allele of:

    • N370S (N409S in recent nomenclature)
    • R496H (R535H in recent nomenclature)
  4. Presence of calcification in heart valves or arteries in echocardiography.
  5. Presence of untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism. (Resolved anemia is not an exclusion criterion.)
  6. Presence of human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and/or hepatitis C infections.
  7. Splenectomy and bone marrow transplantation.
  8. 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.
  9. Any other disorder that may interfere with the results of the efficacy endpoints.
  10. Pregnancy or breastfeeding.
  11. Currently taking another investigational drug for any condition or any therapeutic drug for Gaucher disease.
  12. 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.
  13. Medical history of any food/drugs allergy.

Sites / Locations

  • All India Institute of Medical Sciences
  • Shaare Zedek Medical Center
  • Gazi University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Taliglucerase Alpha

Arm Description

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

Outcomes

Primary Outcome Measures

Percent change from baseline in spleen volume measured by MRI
Percent change from baseline

Secondary Outcome Measures

Percent change from baseline in liver volume measured by MRI
Percent change from baseline
Percent change in hemoglobin
Percent change from baseline
Percent change in platelet count
Percent change from baseline
Percent change in Lyso-GB1
Percent change from baseline

Full Information

First Posted
June 27, 2019
Last Updated
July 21, 2022
Sponsor
Ari Zimran
Collaborators
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT04002830
Brief Title
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Official Title
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Study Type
Interventional

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 20, 2020 (Actual)
Primary Completion Date
July 30, 2023 (Anticipated)
Study Completion Date
August 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Ari Zimran
Collaborators
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

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.
Detailed Description
Patients with Type 3 GD exhibit both visceral and neurologic manifestations. In addition to the progressive neurologic involvement, somatic disease manifestations, especially splenomegaly and resulting cytopenia, contribute to significant mortality and morbidity . The effects of enzyme replacement therapy (ERT) on patients with Type 1 GD have been clearly documented and have a beneficial effect on visceral and hematologic disease parameters . It is known that recombinant enzyme does not pass the blood-brain barrier and has no effect on neurologic involvement . Probably due to the rarity of Type 3 GD, information on the somatic effects of ERT is largely limited to case reports or single-center series. There are also few reviews of cohorts but the clinical subtype, age, genotype, ERT dosage, accompanying therapies, and treatment response vary widely among patients in these cohorts. This prospective study aims to objectively evaluate the hematologic and visceral effects of ERT with taliglucerase alfa on a rather clinically and genetically homogenous group of treatment-naïve patients with Type 3 GD . For the purposes of this study, subjects receiving no Gaucher-specific medications for at least 12 months will be considered "untreated". The results of this study are expected to provide a more objective view of the degree of response of this patient type, and potentially create new areas of research.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease, Type 3

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
Type 3 Gaucher disease patients
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Taliglucerase Alpha
Arm Type
Experimental
Arm Description
Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease
Intervention Type
Drug
Intervention Name(s)
Elelyso
Other Intervention Name(s)
Taliglucerase Alfa
Intervention Description
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.
Primary Outcome Measure Information:
Title
Percent change from baseline in spleen volume measured by MRI
Description
Percent change from baseline
Time Frame
from baseline to month 12
Secondary Outcome Measure Information:
Title
Percent change from baseline in liver volume measured by MRI
Description
Percent change from baseline
Time Frame
from baseline to month 12
Title
Percent change in hemoglobin
Description
Percent change from baseline
Time Frame
from baseline to Months 3, 6, 9, and 12
Title
Percent change in platelet count
Description
Percent change from baseline
Time Frame
from baseline to Months 3, 6, 9, and 12
Title
Percent change in Lyso-GB1
Description
Percent change from baseline
Time Frame
from baseline to Months 3, 6, 9, and 12

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: Eligible subjects may not have any of the following exclusion criteria: 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 untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism. (Resolved anemia is not an exclusion criterion.) 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ari Zimran, Prof.
Organizational Affiliation
Shaare Zedek Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
All India Institute of Medical Sciences
City
New Delhi
Country
India
Facility Name
Shaare Zedek Medical Center
City
Jerusalem
ZIP/Postal Code
9103102
Country
Israel
Facility Name
Gazi University
City
Ankara
Country
Turkey

12. IPD Sharing Statement

Plan to Share IPD
No

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

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