Immune Modulation Therapy for Pompe Disease

Phase 4

• Conditions: Pompe Disease
• Interventions: Drug: Rituximab; Drug: intravenous immune globulin; Drug: Bortezomib; Drug: Methotrexate

• Registration Number: NCT02525172
• Lead Sponsor: National Taiwan University Hospital

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-08
• Study First Submitted: 2015-08-10
• Study First Submitted QC: 2015-08-14
• Study First Posted: 2015-08-17
• Status Verified: 2016-04
• Last Update Submitted: 2016-04-13
• Last Update Posted: 2016-04-15
• Primary Completion: 2020-07
• Study Completion: 2020-07

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 8

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ITT	intravenous immune globulin	immune modulation therapy
ITT	Rituximab	immune modulation therapy
ITT	Methotrexate	immune modulation therapy
ITT	Bortezomib	immune modulation therapy

Primary Outcome Measures

Name	Time	Method
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease	6 months

Trial Locations

Locations (1)

Yin-Hsiu Chien; 🇨🇳 Taipei, Taiwan