Text Size
Trial record 10 of 52 for:    Pompe
Previous Study | Return to List | Next Study

Observational Study for Children With Pompe Disease Undergoing Immune Modulation Therapies

This study is currently recruiting participants.
Verified September 2012 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 21, 2012
Last verified: September 2012
History of Changes
  Purpose

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 children with Pompe.


Condition
Pompe Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Children With Pompe Disease Receiving rhGAA Replacement Therapy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of Florida:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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 - 6 years)


Biospecimen Retention:   Samples With DNA

Primary Skin cell fibroblast line will be maintained and any residual blood samples will be stored as serum and plasma when available.


Estimated Enrollment: 12
Study Start Date: October 2008
Estimated Study Completion Date: October 2018
Estimated Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

  Eligibility

Ages Eligible for Study:   up to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

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

Criteria

Inclusion Criteria:

  • 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, IVIG 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01451879

Contacts
Contact: Lee Ann Lawson, ARNP 352-273-7762 llawson@peds.ufl.edu
Contact: Lindsay Falk, BSN 352-273-9615 lindsayc@peds.ufl.edu

Locations
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Lee Ann Lawson, ARNP     352-273-7762     llawson@pedcard.ufl.edu    
Contact: Lindsay Falk, BSN     352-273-9615     lindsayc@peds.ufl.edu    
Principal Investigator: Barry J. Byrne, MD, PhD            
Sponsors and Collaborators
University of Florida
  More Information

No publications provided

Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT01451879     History of Changes
Other Study ID Numbers: IMN 439-2008
Study First Received: September 15, 2011
Last Updated: September 21, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by University of Florida:
Pompe Disease

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 21, 2013