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A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03227042
Recruitment Status : Active, not recruiting
First Posted : July 24, 2017
Last Update Posted : January 28, 2021
Sponsor:
Information provided by (Responsible Party):
Allievex Corporation

Tracking Information
First Submitted Date July 11, 2017
First Posted Date July 24, 2017
Last Update Posted Date January 28, 2021
Actual Study Start Date November 16, 2017
Estimated Primary Completion Date October 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 19, 2017)
  • Neurocognitive function [ Time Frame: Baseline + every 24 weeks for up to 192 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 192 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 192 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 192 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 192 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.
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 A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Official Title A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Males and Females with a documented diagnosis of MPSIIIB
Condition Mucopolysaccharidosis Type IIIB
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: January 26, 2021)
44
Original Estimated Enrollment
 (submitted: July 19, 2017)
60
Estimated Study Completion Date December 2024
Estimated Primary Completion Date October 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria

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

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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 18 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Argentina,   Australia,   Brazil,   Colombia,   Germany,   Spain,   Taiwan,   Turkey,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03227042
Other Study ID Numbers 250-902
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Undecided
Responsible Party Allievex Corporation
Study Sponsor Allievex Corporation
Collaborators Not Provided
Investigators
Study Director: Medical Director, MD Allievex Medical Monitor
PRS Account Allievex Corporation
Verification Date January 2021