Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by University of Florida
Sponsor:
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT01451879
First received: September 15, 2011
Last updated: September 17, 2014
Last verified: September 2014

September 15, 2011
September 17, 2014
October 2008
October 2018   (final data collection date for primary outcome measure)
Anti-rh GAA antibody titers [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Antibody titer for recombinant alpha acid glucosidase (GAA), or anti-rh-GAA antibody titers will be evaluated at baseline and every 4-8 weeks for 52 weeks of participation in the primary study. Subjects who continue participation in the extension study (>52 weeks - 3 years, anti-rh-GAA antibody titers will be evaluated every 12 weeks
Anti-rh GAA antibody titers [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
anti-rh-GAA antibody titers will be evaluated at baseline and every 4-8 weeks for 52 weeks of participation in the primary study. Subjects who continue participation in the extension study (>52 weeks - 3 years, anti-rh-GAA antibody titers will be evaluated every 12 weeks
Complete list of historical versions of study NCT01451879 on ClinicalTrials.gov Archive Site
Safety Labs [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
Reports from clinical labs including white count, Immune-globulin G (IgG), B-lymphocyte antigen (CD20) will be added to the study record when available every 4-12 weeks during the primary study and every 12 weeks for subjects who participate in the extension study (>52 weeks - 6 years)
Safety Labs [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
Safety labs including white count, IgG, CD20 will be evaluated every 4-12 weeks during the primary study and every 12 weeks for subjects who participate in the extension study (>52 weeks - 3 years)
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Not Provided
 
Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy

The purpose of this research study is to determine the effect(s) of medications that alter the immune system on anti-rhGAA immune response in Pompe patients receiving rhGAA enzyme replacement therapy (ERT), and to understand the immune response in Pompe patients treated with ERT that do not make antibodies against ERT. The investigators would also like to determine whether treating Pompe Disease with medications that affect the immune system has any effects on the overall health or disease progression of Pompe.

Not Provided
Observational
Observational Model: Case-Only
Time Perspective: Prospective
Not Provided
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Non-Probability Sample

The study population will consist of male and female patients age 0-65 years, with a diagnosis of early-onset Pompe Disease. Up to 25 subjects will be enrolled.

Pompe Disease
Not Provided
Age 0 months to 65 years
With a diagnosis of early-onset Pompe Disease. Total of 25 subjects enrolled, some of who will be candidates for immunomodulation therapy.
Elder ME, Nayak S, Collins SW, Lawson LA, Kelley JS, Herzog RW, Modica RF, Lew J, Lawrence RM, Byrne BJ. B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease. J Pediatr. 2013 Apr 16. doi:pii: S0022-3476(13)00267-9. 10.1016/j.jpeds.2013.03.002. [Epub ahead of print]

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
25
October 2018
October 2018   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • patients between the ages of 0 months and 65 years
  • diagnosed with early-onset Pompe Disease, confirmed by mutational analysis and/or GAA enzyme assay
  • eligible regardless of whether they have begun enzyme replacement therapy prior to enrollment
  • all subjects will receive ERT as standard of care during the course of the study, although they may not have begun ERT treatment at the time of enrollment
  • subjects may receive an immunomodulatory regimen as part of their standard of care; this may include rituximab, sirolimus, methotrexate, Gamunex, Hizentra, Zavesca or other immunomodulatory agents, alone or in combination, at the discretion of their caregiver(s)

Exclusion Criteria:

  • subject is unable to meet the study requirements
  • subjects medical condition contraindicates participation or Study Investigators feel that participation is otherwise not in the subject's best interest
  • subject does not receive ERT treatment
Both
up to 65 Years
No
Contact: Lee Ann Lawson, ARNP 352-273-7762 llawson@peds.ufl.edu
United States
 
NCT01451879
IMN 439-2008
No
University of Florida
University of Florida
Not Provided
Principal Investigator: Barry J Byrne, MD, PhD University of Florida
University of Florida
September 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP