Immune Modulation Therapy for Pompe Disease
Primary Purpose
Pompe Disease
Status
Unknown status
Phase
Phase 4
Locations
Taiwan
Study Type
Interventional
Intervention
Rituximab
intravenous immune globulin
Bortezomib
Methotrexate
Sponsored by
About this trial
This is an interventional treatment trial for Pompe Disease focused on measuring immune modulation therapy, acid α-glucosidase, antibody
Eligibility Criteria
Inclusion Criteria:
- The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed;
- The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid α-glucosidase (GAA) enzyme deficiency from blood samples or 2 GAA gene mutations;
- The patient (and/or legal guardian) must have ability to comply with clinical protocol;
- Regimen A only: The patient is receiving enzyme replacement therapy, exhibits clinical decline, and has persistent high anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme;
- Regimen B only: The patient is cross-reactive immune material (CRIM) -negative AND have not received Myozyme infusion prior to enrollment
Exclusion Criteria:
- The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
- The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis;
- The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment;
- The patient is pregnant or lactating;
- The patient has had or is required to have any live vaccination within one month prior to enrollment.
Sites / Locations
- Yin-Hsiu ChienRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ITT
Arm Description
immune modulation therapy
Outcomes
Primary Outcome Measures
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
Secondary Outcome Measures
Full Information
NCT ID
NCT02525172
First Posted
August 10, 2015
Last Updated
April 13, 2016
Sponsor
National Taiwan University Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02525172
Brief Title
Immune Modulation Therapy for Pompe Disease
Official Title
Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients
Study Type
Interventional
2. Study Status
Record Verification Date
April 2016
Overall Recruitment Status
Unknown status
Study Start Date
August 2015 (undefined)
Primary Completion Date
July 2020 (Anticipated)
Study Completion Date
July 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Taiwan University Hospital
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to assess anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers after treatment with immune modulation therapy in patients of Pompe disease.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease
Keywords
immune modulation therapy, acid α-glucosidase, antibody
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ITT
Arm Type
Experimental
Arm Description
immune modulation therapy
Intervention Type
Drug
Intervention Name(s)
Rituximab
Intervention Type
Drug
Intervention Name(s)
intravenous immune globulin
Intervention Type
Drug
Intervention Name(s)
Bortezomib
Intervention Type
Drug
Intervention Name(s)
Methotrexate
Primary Outcome Measure Information:
Title
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
Time Frame
6 months
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed;
The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid α-glucosidase (GAA) enzyme deficiency from blood samples or 2 GAA gene mutations;
The patient (and/or legal guardian) must have ability to comply with clinical protocol;
Regimen A only: The patient is receiving enzyme replacement therapy, exhibits clinical decline, and has persistent high anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme;
Regimen B only: The patient is cross-reactive immune material (CRIM) -negative AND have not received Myozyme infusion prior to enrollment
Exclusion Criteria:
The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis;
The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment;
The patient is pregnant or lactating;
The patient has had or is required to have any live vaccination within one month prior to enrollment.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yin-Hsiu Chien
Phone
+886223123456
Email
chienyh@ntu.edu.tw
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Yin-Hsiu Chien
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien
Phone
+886223123456
Email
chienyh@ntu.edu.tw
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien
12. IPD Sharing Statement
Learn more about this trial
Immune Modulation Therapy for Pompe Disease
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