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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03227042




Registration number
NCT03227042
Ethics application status
Date submitted
11/07/2017
Date registered
24/07/2017
Date last updated
26/10/2022

Titles & IDs
Public title
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Scientific title
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Secondary ID [1] 0 0
250-902
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis Type IIIB 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Neurocognitive function
Timepoint [1] 0 0
Baseline + every 24 weeks for up to 240 weeks
Primary outcome [2] 0 0
Behavioral function
Timepoint [2] 0 0
Baseline + every 24 weeks for up to 240 weeks
Primary outcome [3] 0 0
Quality of Life Tests
Timepoint [3] 0 0
Baseline + every 24 weeks for up to 240 weeks
Primary outcome [4] 0 0
Sleep habits
Timepoint [4] 0 0
Baseline + every 24 weeks for up to 240 weeks
Primary outcome [5] 0 0
Disease-specific Biomarkers
Timepoint [5] 0 0
Baseline + every 24 weeks for up to 240 weeks
Primary outcome [6] 0 0
Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden
Timepoint [6] 0 0
Once (at baseline visit)

Eligibility
Key inclusion criteria
* 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
Minimum age
No limit
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/11/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
Accrual to date
Final
44
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Childrens Research Institute and Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
Argentina
State/province [2] 0 0
Pilar
Country [3] 0 0
Brazil
State/province [3] 0 0
Rio Grande Do Sul
Country [4] 0 0
Colombia
State/province [4] 0 0
Bogotá
Country [5] 0 0
Germany
State/province [5] 0 0
Hamburg
Country [6] 0 0
Spain
State/province [6] 0 0
A Coruña
Country [7] 0 0
Taiwan
State/province [7] 0 0
Taipei
Country [8] 0 0
Turkey
State/province [8] 0 0
Ankara

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Allievex Corporation
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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. 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.
Trial website
https://clinicaltrials.gov/study/NCT03227042
Trial related presentations / 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.
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
Allievex Medical Monitor
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03227042