MPS II Immunophenotyping

study id #: NCT04976231

condition: MPS II, Mucopolysaccharidosis II, Hunter Syndrome

status: Not yet recruiting

The purpose of this study is to investigate how participant’s body’s immune system responds to idursulfase, an enzyme replacement therapy (ERT) and find out which types of immune cells are involved in causing untoward responses to the ERT so that the investigators can relate the level of immune response to the treatment.

results: https://clinicaltrials.gov/ct2/show/results/NCT04976231

last updated: February 26, 2022

start date: March 2022

estimated completion: July 31, 2023

last updated: January 27, 2022

size / enrollment: 25

primary outcomes:

• Changes in the frequency of natural killer cells measured by flow cytometry
  
• baseline, 1, 3, 6 months
  Changes in level of circulation memory B cells measured by flow cytometry
  
• baseline, 1, 3, 6 months
  Changes in level of T follicular helper cells measured by flow cytometry
  
• baseline, 1, 3, 6 months
  Changes in distribution of helper T cells measured by flow cytometry
  
• baseline, 1, 3, 6 months
  

inclusion criteria:

exclusion criteria: Criteria:

None

sponsor: Duke University

contacts: Lauren Workman, 919 660-0758, lauren.workman@duke.edu

investigators: Priya Kishnani, MD,Duke University

trial center locations: