Who is sponsoring NCT01451879?

NCT01451879 is sponsored by University of Florida.

What condition does NCT01451879 study?

NCT01451879 studies Pompe Disease.

What drugs are being tested in NCT01451879?

Interventions: Rituximab, Miglustat.

What is the status of NCT01451879?

NCT01451879 is currently COMPLETED.

Last reviewed 2017-12-06 · How we verify

Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy

NCT01451879 COMPLETED

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.

Details

Lead sponsor	University of Florida
Status	COMPLETED
Enrolment	11
Start date	2008-10
Completion	2017-10

Conditions

Pompe Disease

Interventions

Rituximab
Miglustat

Primary outcomes

Anti-rh GAA antibody titers — 52 weeks
Antibody titer for anti-rh-GAA will be evaluated at baseline and every 4-8 weeks for 52 weeks of participation in the primary study. For subjects who continue participation in the extension study (\>52 weeks - 5 years), anti-rh-GAA antibody titers will be evaluated every 12 - 24 weeks.

Countries

United States