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出境医 / 临床实验 / A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

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

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.

Condition or disease Intervention/treatment Phase
Gaucher Disease, Type 3 Drug: Elelyso Phase 4

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.
Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Type 3 Gaucher disease patients
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Actual Study Start Date : November 20, 2020
Estimated Primary Completion Date : August 1, 2022
Estimated Study Completion Date : August 1, 2022
Arms and Interventions
Arm Intervention/treatment
Experimental: Taliglucerase Alpha
Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease
 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.
Other Name: Taliglucerase Alfa
Outcome Measures
Primary Outcome Measures :
  1. Percent change from baseline in spleen volume measured by MRI [ Time Frame: from baseline to month 12 ]
    Percent change from baseline


Secondary Outcome Measures :
  1. Percent change from baseline in liver volume measured by MRI [ Time Frame: from baseline to month 12 ]
    Percent change from baseline

  2. Percent change in hemoglobin [ Time Frame: from baseline to Months 3, 6, 9, and 12 ]
    Percent change from baseline

  3. Percent change in platelet count [ Time Frame: from baseline to Months 3, 6, 9, and 12 ]
    Percent change from baseline

  4. Percent change in Lyso-GB1 [ Time Frame: from baseline to Months 3, 6, 9, and 12 ]
    Percent change from baseline


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations

Contacts
Layout table for location contacts
Contact: Majdoleen J Istaiti, B.Sc +972-52-6659995 joleenist@szmc.org.il

Locations
Layout table for location information
India
All India Institute of Medical Sciences Recruiting
New Delhi, India
Contact: Madhulika Kabra, M.D       madhulikakabra@hotmail.com   
Israel
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel, 9103102
Contact: Majdolen J Istaiti, B.Sc    +97-52-6659995    joleenist@szmc.org.il   
Principal Investigator: Ari Zimran, Prof         
Sub-Investigator: Shoshana Revel-Vilk, Prof         
Turkey
Gazi University Not yet recruiting
Ankara, Turkey
Contact: Fatih Ezgu       fatih.ezgu@gmail.com   
Sponsors and Collaborators
Ari Zimran
Pfizer
Investigators
Layout table for investigator information
Principal Investigator: Ari Zimran, Prof. Shaare Zedek Medical Center
Tracking Information
First Submitted Date  ICMJE June 27, 2019
First Posted Date  ICMJE July 1, 2019
Last Update Posted Date March 19, 2021
Actual Study Start Date  ICMJE November 20, 2020
Estimated Primary Completion Date August 1, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 27, 2019) 		Percent change from baseline in spleen volume measured by MRI [ Time Frame: from baseline to month 12 ]
Percent change from baseline
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 27, 2019)
  • Percent change from baseline in liver volume measured by MRI [ Time Frame: from baseline to month 12 ]
    Percent change from baseline
  • Percent change in hemoglobin [ Time Frame: from baseline to Months 3, 6, 9, and 12 ]
    Percent change from baseline
  • Percent change in platelet count [ Time Frame: from baseline to Months 3, 6, 9, and 12 ]
    Percent change from baseline
  • Percent change in Lyso-GB1 [ Time Frame: from baseline to Months 3, 6, 9, and 12 ]
    Percent change from baseline
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Official Title  ICMJE A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Type 3 Gaucher disease patients
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Gaucher Disease, Type 3
Intervention  ICMJE 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.
Other Name: Taliglucerase Alfa
Study Arms  ICMJE Experimental: Taliglucerase Alpha
Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease
Intervention: Drug: Elelyso
Publications * Not Provided

*   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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 27, 2019) 		15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 1, 2022
Estimated Primary Completion Date August 1, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Majdoleen J Istaiti, B.Sc +972-52-6659995 joleenist@szmc.org.il
Listed Location Countries  ICMJE India,   Israel,   Turkey
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04002830
Other Study ID Numbers  ICMJE WI224302
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Ari Zimran, Shaare Zedek Medical Center
Study Sponsor  ICMJE Ari Zimran
Collaborators  ICMJE Pfizer
Investigators  ICMJE
Principal Investigator: Ari Zimran, Prof. Shaare Zedek Medical Center
PRS Account Shaare Zedek Medical Center
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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