Natural History Study to Characterise the Course of Disease Progression in Participants With Muco… (NCT02293408) | Clinical Trial Compass
TerminatedNot Applicable
Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB
Stopped: The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.
United States, Brazil, Italy30 participantsStarted 2014-09-14
Plain-language summary
The objectives of this study are to describe the clinical and biochemical characteristics and course of disease progression in participants with Mucopolysaccharidosis type IIIB (MPS IIIB)
Who can participate
Age range1 Year
SexALL
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Inclusion criteria
✓. The participant has a definitive diagnosis of MPS IIIB, as determined by either of the following:
✓. Documented deficiency in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity or
✓. Documented functionally-relevant mutations in both alleles of the NAGLU gene.
✓. The participant is at least 1 year of age (biological age).
✓. The participant or the participant's parent provides informed consent.
✓. The participant is willing and able to comply with protocol requirements to the extent that may be expected of a participant with cognitive impairment.
✓. The participant meets criterion a or criterion b below.
✓. The participant has an age equivalent of ≥1 year on the Vineland Adaptive Behavior Scales, Second Edition (Vineland II).
Exclusion criteria
✕. The participant has visual or hearing impairments sufficient to preclude cooperation with neurodevelopmental testing.
✕. The participant has a history of poorly-controlled seizure disorder.
✕. The participant is currently receiving medication, which, in the Investigator's opinion, would be likely to substantially confound interpretation of the results (for example, the participant has been on the current dose of psychotropic medication for less than 3 months).
What they're measuring
1
Collection And Analysis Of Clinical Characteristics Of MPS IIIB
Timeframe: Baseline to Week 43
2
Longitudinal Analysis Of The Course Of Disease Progression In Participants With MPS IIIB
. The participant is receiving a newly increased dose of melatonin (for example, less than 3 months on current dose).
✕. The participant has previously received an investigational therapy for MPS IIIB (with the exception of high dose Genistein \>150 milligram/kilogram (mg/kg)/day, which will require a minimum of 3 months wash-out before entering the study) or has had hematopoietic stem cell transplant (HSCT).
✕. The participant has any other prior or ongoing medical condition that may present a safety risk, interfere with study compliance, or confound data interpretation.