Other Clinical Trial - Natural History Observational Study of MPS IIIa in

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

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.

NCT number	NCT04918641
Study type	Observational [Patient Registry]
Source	Samsung Medical Center
Contact	SungYoon Cho
Phone	+82-2-3410-3537
Email	nadri1217@naver.com
Status	Not yet recruiting
Phase
Start date	June 30, 2021
Completion date	July 28, 2024

Natural History Observational Study of MPS IIIa in SMC

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms