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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.
Full 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.
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Inclusion and exclusion criteria
Inclusion Criteria:
Each patient must meet all of the following criteria to be enrolled in this study.
Definitive diagnosis of MPS IIIB, as determined by one of the following:
The patient is ≥1 and < 10 years of age and has an age equivalent on the Vineland Adaptive Behavior Scales (VABS) of ≥1 year
The patient is medically stable, in the opinion of the Investigator, to accommodate the protocol requirements, including travel and assessments, without placing an undue burden on the patient/patient's family.
The patient, patient's parent(s) or legal guardian(s) has voluntarily signed and dated an Institutional Review Board/Independent Ethics Committee-approved informed consent (assent if applicable) form after all relevant aspects of the study have been explained and discussed with the patient, the patient's parent(s), or legal guardian(s).Exclusion Criteria:
Patients will be excluded from the study if they meet any of the following criteria at screening:
19 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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