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COMPLETED

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT01509768 · View on ClinicalTrials.gov ↗

Study 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.

Conditions Studied

Study Locations (4)

Other

  • Johannes Gutenberg University Medical Center — Mainz
  • Great Ormond Street Hospital for Children — London

California

  • Children's Hospital and Research Center — Oakland

Minnesota

  • University of Minnesota — Minneapolis

Trial Details

FieldValue
Enrollment Target 19 participants
Start Date 2012-04-09
Est. Completion 2013-10-10

Sponsor

Shire

56 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT01509768

The ClinicalTrials.gov registry entry for NCT01509768 describes a study currently listed as completed. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 19 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Shire, which has 56 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 1 condition, with Sanfilippo Syndrome Type B appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT01509768 reports 4 study locations spanning 3 distinct geographic areas — top geographies include Other, California, Minnesota. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT01509768 about?

NCT01509768 is a clinical study titled "Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)". 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 stan...

What is the current status of trial NCT01509768?

This trial is currently completed. The enrollment target is 19 participants. The study started on 2012-04-09. Estimated completion is 2013-10-10.

What conditions does trial NCT01509768 study?

This clinical trial studies the following conditions: Sanfilippo Syndrome Type B. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

Who is sponsoring clinical trial NCT01509768?

This trial is sponsored by Shire, which has 56 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT01509768 being conducted?

This trial has 4 study locations across California, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial