A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Clinical Trial Summary

The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.

See also
	Status	Clinical Trial	Phase
	Completed	`NCT01873911` - Neurobehavioral Phenotypes in MPS III

NCT number	NCT01047306
Study type	Observational
Source	Takeda
Contact
Status	Completed
Phase
Start date	February 15, 2010
Completion date	July 10, 2013

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms