Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Part of paid clinical trials in Gainesville, Florida.

Sponsor
University of Florida
Study ID
NCT01451879
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Rituximab — DRUG
    Clinically prescribed immune modulation regimen dosage determined by local medical provider.
  • Miglustat — DRUG
    Clinically prescribed immune modulation regimen dosage determined by local medical provider.

Study Details

Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme. Treatment with immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Studying the immune response to rhGAA may provide valuable insight into the role of the immune system in the effectiveness of ERT for Pompe Disease.

Key Dates

Start date
Oct 31, 2008
Status verified
Dec 2017
Primary completion
Oct 31, 2017
Completion
Oct 31, 2017

Study Design

Enrollment
11 participants (actual)

Arms

  • Arm: Age 0 months to 65 years
    Confirmed diagnosis of Pompe Disease, and clinically prescribed immune modulation regimen with agents such as rituximab, sirolimus, methotrexate, IVIg or other immunomodulatory agents such as pharmacological chaperone Miglustat, N-butyldeoxynojirimycin (NB-DNJ), alone or in combination, at the discretion of their primary/specialist caregiver.

Primary Outcome Measure

Anti-rh GAA antibody titers [ Time Frame: 52 weeks ]

Locations (1)

FacilityCityStateZIPSite coordinators
University of FloridaGainesvilleFlorida32610-

Find similar trials in Gainesville, FL

By research site
University of Florida· Gainesville, FL

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