MPS IIIB (Sanfilippo Syndrome)NCT02293382
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
An observational study of MPS IIIB (Sanfilippo Syndrome), run by Alexion Pharmaceuticals, Inc. at 5 sites in 5 countries.
Trial summary
The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MPS IIIB (Sanfilippo Syndrome)
- How many people
- 30 participants
- Where
- 5 sitesin 5 countries
- Study dates
- Jul 2013to Jan 2015
- 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: 30 deceased patients with MPS IIIB
Who may be able to join
- The deceased patient was diagnosed with MPS IIIB as determined by either of the following:
- Documented deficiency of NAGLU enzyme activity.
- Documented functionally-relevant mutations in both alleles of the NAGLU gene.
- Patient's parent or legal guardian provides informed consent (unless the requirement for informed consent is waived by local regulations).
- The availability of pre-defined information in the patient's clinical chart:
Who cannot join
- There are no exclusion criteria for this study.
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
About this study
The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.
- Conditions
- MPS IIIB (Sanfilippo Syndrome)
- Keywords
- MPS IIIB, Mucopolysaccharodosis III, Mucopolysaccharidosis type IIIB, Sanfilippo Syndrome
- Medical subject headings
- Mucopolysaccharidosis III
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients.
Data obtained by retrospective chart review will include medical history data including patient demographics, disease manifestation onset, clinical history, diagnostic tests and treatments for MPS IIIB, supportive interventions/medications, clinical chemistry and haematology results, anthropometric data, radiology results, and autopsy findings.
Measured: N/A as this is a retrospective chart review of deceased patients
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2014 (estimated)
- Study startJul 2013
- Primary completionJan 2015
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS IIIB (Sanfilippo Syndrome)Studies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02293382
- Sponsor's protocol ID
- NGLU-NH01
- Status last verified
- Feb 2017
- First submitted
- 3 Nov 2014
- Last updated
- 3 Feb 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
- ClinicalTrials.gov ID
- NCT02293382
- Acronym / sponsor ID
- NGLU-NH01
- Conditions
- MPS IIIB (Sanfilippo Syndrome)
- Keywords
- MPS IIIB, Mucopolysaccharodosis III, Mucopolysaccharidosis type IIIB, Sanfilippo Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2013
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 18 Nov 2014 (estimated)
- 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 objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 30 deceased patients with MPS IIIB
Key inclusion criteria (5 listed)
- The deceased patient was diagnosed with MPS IIIB as determined by either of the following:
- Patient's parent or legal guardian provides informed consent (unless the requirement for informed consent is waived by local regulations).
- The availability of pre-defined information in the patient's clinical chart:
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study.
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
Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients.
Time frame: N/A as this is a retrospective chart review of deceased patients
Data obtained by retrospective chart review will include medical history data including patient demographics, disease manifestation onset, clinical history, diagnostic tests and treatments for MPS IIIB, supportive interventions/medications, clinical chemistry and haematology results, anthropometric data, radiology results, and autopsy findings.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2017.
Related trials for MPS IIIB (Sanfilippo Syndrome)
Active, not recruitingPhase 1/2
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Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
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Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
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ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
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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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of MPS IIIB (Sanfilippo Syndrome), run by Alexion Pharmaceuticals, Inc. at 5 sites in 5 countries.
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 5 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: finished, as of the sponsor's last update on 3 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Brazil, Netherlands, Spain, United Kingdom. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients. (time frame: N/A as this is a retrospective chart review of deceased patients). 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 3 Feb 2017.