Immune Modulation Therapy for Pompe Disease

Sponsor
National Taiwan University Hospital
Study ID
NCT02525172
Phase
PHASE4
Status
Unknown

Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Rituximab — DRUG
  • intravenous immune globulin — DRUG
  • Bortezomib — DRUG
  • Methotrexate — DRUG

Study Details

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.

Key Dates

Start date
Aug 31, 2015
Status verified
Apr 2016
Primary completion
Jul 31, 2020
Completion
Jul 31, 2020

Study Design

Enrollment
8 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: ITT
    immune modulation therapy

Primary Outcome Measure

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

Central Contacts

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