Mucopolysaccharidosis Type IIIBNCT03227042
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
An observational study of Mucopolysaccharidosis Type IIIB, run by Allievex Corporation at 9 sites in 9 countries.
Trial summary
This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Mucopolysaccharidosis Type IIIB
- How many people
- 44 participants
- Where
- 9 sitesin 9 countries
- Study dates
- 16 Nov 2017to Dec 2025
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Males and Females with a documented diagnosis of MPSIIIB
Who may be able to join
- Have deficient NAGLU enzyme activity at Baseline. Blood for NAGLU enzyme activity will be collected and analyzed centrally.
- Is up to 18 years of age
- Written informed consent from parent or legal guardian and assent from subject, if required
- Has the ability to comply with protocol requirements, in the opinion of the investigator
Who cannot join
- Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry
- Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB
- Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study
- Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's wellbeing or safety, or the interpretability of the subject's clinical data.
- Is currently participating in another natural history study
Trial locations and contacts
9 sites in 9 countries.
9 sites in 9 countries
United States1 site
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Australia1 site
Murdoch Childrens Research Institute and Royal Children's Hospital
Melbourne, Victoria, 3052
Brazil1 site
Medical Genetics Service/HCPA, Department of Genetics/UFRGS
Pôrto Alegre, Rio Grande do Sul, 90035-903
Spain1 site
Hospital Clínico Universitario de Santiago
Santiago de Compostela, A Coruña, 15706
Turkey (Türkiye)1 site
Gazi University Faculty of Medicine
Ankara, 06560
About this study
This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by Allievex in this study.
- Conditions
- Mucopolysaccharidosis Type IIIB
- Keywords
- Sanfilippo Syndrome Type B, MPS IIIB, MPS 3 B
- Medical subject headings
- Mucopolysaccharidosis III
What is being measured
The primary outcome is the main question the study is designed to answer.
Neurocognitive function
A neurodevelopmental assessment will be performed using standardized developmental tests to provide quantifiable measures of neurocognitive function.
Measured: Baseline + every 24 weeks for up to 240 weeks
Behavioral function
Disease-related behaviors will be assessed using an MPS IIIB specific behavior rating scale.
Measured: Baseline + every 24 weeks for up to 240 weeks
Quality of Life Tests
Multiple QoL tools will be used to capture physical, mental, and social well-being of the patient as well as to examine the impact of the patient's disease on the parent/guardian and family.
Measured: Baseline + every 24 weeks for up to 240 weeks
Sleep habits
Patient sleep habits will be assessed using Children's Sleep Habits Questionnaires (CSHQ).
Measured: Baseline + every 24 weeks for up to 240 weeks
Disease-specific Biomarkers
Urine sample for glycosaminoglycans (GAGs) and creatinine.
Measured: Baseline + every 24 weeks for up to 240 weeks
Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden
Blood and urine samples will be used to evaluate biochemical, molecular cellular, and genetic/genomic aspects of MPS IIIB.
Measured: Once (at baseline visit)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2017
- Study start16 Nov 2017
- Primary completionOct 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis Type IIIBStudies of the same condition, open and under way.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Allievex Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Allievex Medical Monitor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03227042
- Sponsor's protocol ID
- 250-902
- Status last verified
- Oct 2022
- First submitted
- 11 Jul 2017
- Last updated
- 26 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Okur I, Ezgu F, Giugliani R, Muschol N, Koehn A, Amartino H, Harmatz P, de Castro Lopez MJ, Couce ML, Lin SP, Batzios S, Cleary M, Solano M, Peters H, Lee J, Nestrasil I, Shaywitz AJ, Maricich SM, Kuca B, Kovalchin J, Zanelli E. Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB. J Pediatr. 2022 Oct;249:50-58.e2. doi: 10.1016/j.jpeds.2022.06.005. Epub 2022 Jun 13.
PubMed 35709957
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
- ClinicalTrials.gov ID
- NCT03227042
- Acronym / sponsor ID
- 250-902
- Conditions
- Mucopolysaccharidosis Type IIIB
- Keywords
- Sanfilippo Syndrome Type B, MPS IIIB, MPS 3 B
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Allievex CorporationIndustry (pharma, biotech, devices)
- Study start
- 16 Nov 2017 (actual)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 24 Jul 2017 (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.
This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by Allievex in this study.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Males and Females with a documented diagnosis of MPSIIIB
Key inclusion criteria (4 listed)
- Have deficient NAGLU enzyme activity at Baseline. Blood for NAGLU enzyme activity will be collected and analyzed centrally.
- Is up to 18 years of age
- Written informed consent from parent or legal guardian and assent from subject, if required
- Has the ability to comply with protocol requirements, in the opinion of the investigator
Key exclusion criteria (5 listed)
- Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry
- Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB
- Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study
- Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's wellbeing or safety, or the interpretability of the subject's clinical data.
- Is currently participating in another natural history 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
Neurocognitive function
Time frame: Baseline + every 24 weeks for up to 240 weeks
A neurodevelopmental assessment will be performed using standardized developmental tests to provide quantifiable measures of neurocognitive function.
Behavioral function
Time frame: Baseline + every 24 weeks for up to 240 weeks
Disease-related behaviors will be assessed using an MPS IIIB specific behavior rating scale.
Quality of Life Tests
Time frame: Baseline + every 24 weeks for up to 240 weeks
Multiple QoL tools will be used to capture physical, mental, and social well-being of the patient as well as to examine the impact of the patient's disease on the parent/guardian and family.
Sleep habits
Time frame: Baseline + every 24 weeks for up to 240 weeks
Patient sleep habits will be assessed using Children's Sleep Habits Questionnaires (CSHQ).
Disease-specific Biomarkers
Time frame: Baseline + every 24 weeks for up to 240 weeks
Urine sample for glycosaminoglycans (GAGs) and creatinine.
Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden
Time frame: Once (at baseline visit)
Blood and urine samples will be used to evaluate biochemical, molecular cellular, and genetic/genomic aspects of MPS IIIB.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2022.
- Derived from this study: PubMed 35709957Okur I, Ezgu F, Giugliani R, Muschol N, Koehn A, Amartino H, Harmatz P, de Castro Lopez MJ, Couce ML, Lin SP, Batzios S, Cleary M, Solano M, Peters H, Lee J, Nestrasil I, Shaywitz AJ, Maricich SM, Kuca B, Kovalchin J, Zanelli E. Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB. J Pediatr. 2022 Oct;249:50-58.e2. doi: 10.1016/j.jpeds.2022.06.005. Epub 2022 Jun 13.
- Individual participant dataSharing planned: Undecided
Related trials for Mucopolysaccharidosis Type IIIB
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RecruitingPhase 1/2
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Mucopolysaccharidosis IIIB
Not yet recruitingPhase 3
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
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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.
Medical Director, MD study director
Allievex Medical Monitor
9 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis Type IIIB, run by Allievex Corporation at 9 sites in 9 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 4 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 26 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Argentina, Australia, Brazil, Colombia and 4 more countries. 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?
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?
Neurocognitive function (time frame: Baseline + every 24 weeks for up to 240 weeks). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03227042, which also lists 1 linked publication.
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 26 Oct 2022.