Immune Modulation Therapy for Pompe Disease

Pompe Disease Clinical Trial

Official title:

Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02525172
• Other study ID #: 201504036MIPB
• Status: Recruiting
• Phase: Phase 4
• First received: August 10, 2015
• Last updated: April 13, 2016
• Start date: August 2015
• Est. completion date: July 2020

Study information

• Verified date: April 2016
• Source: National Taiwan University Hospital
• Contact: Yin-Hsiu Chien
• Phone: +886223123456
• Email: chienyh[@]ntu.edu.tw
• Is FDA regulated: No
• Health authority: Taiwan: Ministry of Health and Welfare
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 8
• Est. completion date: July 2020
• Est. primary completion date: July 2020
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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 a-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 a-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

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease

Intervention

Drug:
• Rituximab

• intravenous immune globulin

• Bortezomib

• Methotrexate

Locations

Country	Name	City	State
Taiwan	Yin-Hsiu Chien	Taipei

Sponsors (1)

Lead Sponsor	Collaborator
National Taiwan University Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	anti-recombinant human acid a-glucosidase (anti-rhGAA) antibody titers decrease		6 months	No