MPS IINCT04976231
MPS II Immunophenotyping
An observational study of MPS II and Mucopolysaccharidosis II, run by Duke University at 1 site in United States.
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Apr 2022to 26 Apr 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Metabolic or Genetics Clinic patients
Who may be able to join
- 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.
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy
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.
- Conditions
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Keywords
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Medical subject headings
- Sudden Infant Death, Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Naive | Subjects who are naïve to ERT and start treatment with idursulfase | |
| Treatment less than 3 years | Subjects who have received ERT for <3 years | |
| Treatment over 3 years | Subjects who have received ERT for 3 or more years |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in the frequency of natural killer cells measured by flow cytometry
Measured: baseline, 1, 3, 6 months
Changes in level of circulation memory B cells measured by flow cytometry
Measured: baseline, 1, 3, 6 months
Changes in level of T follicular helper cells measured by flow cytometry
Measured: baseline, 1, 3, 6 months
Changes in distribution of helper T cells measured by flow cytometry
Measured: baseline, 1, 3, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2021
- Study start1 Apr 2022
- Primary completion26 Apr 2024
- Study completion26 Apr 2024
Why it stopped: Low Enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS IIStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Priya Kishnani, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04976231
- Sponsor's protocol ID
- Pro00108243
- Status last verified
- Apr 2024
- First submitted
- 19 Jul 2021
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT04976231
- Acronym / sponsor ID
- Pro00108243
- Conditions
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Keywords
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Low Enrollment
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- 1 Apr 2022 (actual)
- Primary completion
- 26 Apr 2024 (actual)
- Study completion
- 26 Apr 2024 (actual)
- First posted
- 26 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Treatment Naive | – | –Subjects who are naïve to ERT and start treatment with idursulfase |
| Treatment less than 3 years | – | –Subjects who have received ERT for <3 years |
| Treatment over 3 years | – | –Subjects who have received ERT for 3 or more years |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Metabolic or Genetics Clinic patients
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Changes in the frequency of natural killer cells measured by flow cytometry
Time frame: baseline, 1, 3, 6 months
Changes in level of circulation memory B cells measured by flow cytometry
Time frame: baseline, 1, 3, 6 months
Changes in level of T follicular helper cells measured by flow cytometry
Time frame: baseline, 1, 3, 6 months
Changes in distribution of helper T cells measured by flow cytometry
Time frame: baseline, 1, 3, 6 months
Results and safety
The study was stopped early (reason given: Low Enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: No
Related trials for MPS II
Active, not recruitingPhase 3
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
Mucopolysaccharidosis II
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome
RecruitingPhase 1/2
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
Mucopolysaccharidosis IIIB
Active, not recruitingPhase 2/3
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Mucopolysaccharidosis Type II, Metabolic Diseases
Not yet recruitingPhase 1/2
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
Sanfilippo Syndrome (MPS III)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Priya Kishnani, MD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of MPS II and Mucopolysaccharidosis II, run by Duke University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Low Enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in the frequency of natural killer cells measured by flow cytometry (time frame: baseline, 1, 3, 6 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.