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NCT04976231 Clinical Trial

MPS II Immunophenotyping

Mucopolysaccharidosis II Clinical Trial

Official title:
Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT04976231
Other study ID # Pro00108243
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date April 1, 2022
Est. completion date April 2025

Study information
Verified date April 2024
Source Duke University
Contact Grace Stafford, BS
Phone 919-668-1347
Email grace.stafford@duke.edu
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 25
Est. completion date April 2025
Est. primary completion date April 2025
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: - Subject has a confirmed diagnosis of Mucopolysaccharidosis type II (MPS II) or Hunter Syndrome based on enzyme activity and variant analysis. - Subject who plan to receive or have received enzyme replacement therapy with idursulfase. Exclusion Criteria: - None

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
United States Duke University Durham North Carolina

Sponsors (1)
Lead Sponsor Collaborator
Duke University

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Changes in the frequency of natural killer cells measured by flow cytometry baseline, 1, 3, 6 months
Primary Changes in level of circulation memory B cells measured by flow cytometry baseline, 1, 3, 6 months
Primary Changes in level of T follicular helper cells measured by flow cytometry baseline, 1, 3, 6 months
Primary Changes in distribution of helper T cells measured by flow cytometry baseline, 1, 3, 6 months
See also
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Completed NCT03529786 - Mucopolysaccharidosis Type II Natural History
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Terminated NCT01675674 - Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics N/A
Enrolling by invitation NCT06075537 - An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 Phase 2/Phase 3
Recruiting NCT05422482 - A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ Phase 1
Completed NCT00069641 - Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Phase 2/Phase 3
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Active, not recruiting NCT04348136 - An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II Phase 2/Phase 3
Completed NCT04007536 - A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
Completed NCT00004454 - Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) Phase 1/Phase 2
Active, not recruiting NCT04628871 - Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Terminated NCT00748969 - Clinical Trial of Growth Hormone in MPS I, II, and VI Phase 2/Phase 3
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Completed NCT01301898 - To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients Phase 1/Phase 2
Terminated NCT03041324 - Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II Phase 1/Phase 2
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT03128593 - A Study of JR-141 in Patients With Mucopolysaccharidosis Type II Phase 1/Phase 2
Withdrawn NCT04591834 - Mucopolysaccharidosis Type II Observational
Enrolling by invitation NCT04597385 - Long-term Follow-Up for RGX-121