Immune Modulation Therapy for Pompe Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 25 April 2016
• Main ID: NCT02525172
• Date of registration: 10/08/2015
• Prospective Registration: No
• Primary sponsor: National Taiwan University Hospital
• Public title: Immune Modulation Therapy for Pompe Disease
• Scientific title: Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients
• Date of first enrolment: August 2015
• Target sample size: 8
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02525172
• Study type: Interventional
• Study design: Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 4

Countries of recruitment

Taiwan

Contacts

• Name: Yin-Hsiu Chien

• Name: Yin-Hsiu Chien
• Affiliation: National Taiwan University Hospital

• Name: Yin-Hsiu Chien

Key inclusion & exclusion 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 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.



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Drug: Methotrexate

Drug: Rituximab

Drug: Bortezomib

Drug: intravenous immune globulin

Primary Outcome(s)

anti-recombinant human acid a-glucosidase (anti-rhGAA) antibody titers decrease [Time Frame: 6 months]

Secondary ID(s)

201504036MIPB

Source(s) of Monetary Support

Please refer to primary and secondary sponsors