Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
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ClinicalTrials.gov Identifier: NCT01509768 |
Recruitment Status :
Completed
First Posted : January 13, 2012
Last Update Posted : March 17, 2021
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Sponsor:
Shire
Information provided by (Responsible Party):
Takeda ( Shire )
Tracking Information | ||||
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First Submitted Date | January 10, 2012 | |||
First Posted Date | January 13, 2012 | |||
Last Update Posted Date | March 17, 2021 | |||
Actual Study Start Date | April 9, 2012 | |||
Actual Primary Completion Date | October 10, 2013 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures |
evaluate the course of disease progression in patients with MPS IIIB who are untreated with any investigational products to inform possible future treatment studies [ Time Frame: 13 months ] | |||
Original Primary Outcome Measures | Same as current | |||
Change History | ||||
Current Secondary Outcome Measures | Not Provided | |||
Original Secondary Outcome Measures | Not Provided | |||
Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title | Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | |||
Official Title | An Observational Prospective Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | |||
Brief Summary | The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future treatment trials of MPS IIIB using predefined assessments including standardized clinical, biochemical, neurocognitive, developmental, and imaging measures. | |||
Detailed Description | This is a multicenter longitudinal, prospective, natural history study of patients with MPSIIIB designed to identify potential surrogate endpoints that may be utilized in future Enzyme replacement therapy (ERT) trials of MPS IIIB via defined assessments including standardized clinical, biochemical, neurocognitive, development, and imaging measures. | |||
Study Type | Observational | |||
Study Design | Observational Model: Cohort Time Perspective: Prospective |
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Target Follow-Up Duration | Not Provided | |||
Biospecimen | Retention: Samples With DNA Description: blood (plasma and serum), cerebrospinal fluid (CSF), urine
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Sampling Method | Non-Probability Sample | |||
Study Population | Patients with a documented diagnosis of MPS IIIB and who are currently untreated with investigational products (drugs/device) for this disease. Patient must also be ≥ 1 year and < 10 years of age with an age equivalent on the Vineland Adaptive Behavior Scales (VABS) of ≥ 1 year. | |||
Condition | Sanfilippo Syndrome Type B | |||
Intervention | Not Provided | |||
Study Groups/Cohorts | No treatment
This is a longitudinal, prospective, observational, natural history study of patients with MPS IIIB to identify endpoints that may be used for future ERT trials via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures
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Publications * | Whitley CB, Cleary M, Eugen Mengel K, Harmatz P, Shapiro E, Nestrasil I, Haslett P, Whiteman D, Alexanderian D. Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B. J Pediatr. 2018 Jun;197:198-206.e2. doi: 10.1016/j.jpeds.2018.01.044. Epub 2018 Apr 13. | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status | Completed | |||
Actual Enrollment |
19 | |||
Original Estimated Enrollment |
25 | |||
Actual Study Completion Date | October 10, 2013 | |||
Actual Primary Completion Date | October 10, 2013 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria | Inclusion Criteria: Each patient must meet all of the following criteria to be enrolled in this study.
Patients will be excluded from the study if they meet any of the following criteria at screening:
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Sex/Gender |
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Ages | 1 Year to 10 Years (Child) | |||
Accepts Healthy Volunteers | No | |||
Contacts | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries | Germany, United Kingdom, United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number | NCT01509768 | |||
Other Study ID Numbers | HGT-SNB-088 | |||
Has Data Monitoring Committee | No | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement | Not Provided | |||
Current Responsible Party | Takeda ( Shire ) | |||
Original Responsible Party | Shire Human Genetic Therapies, Inc. | |||
Current Study Sponsor | Shire | |||
Original Study Sponsor | Shire Human Genetic Therapies, Inc. | |||
Collaborators | Not Provided | |||
Investigators |
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PRS Account | Takeda | |||
Verification Date | March 2021 |