A Treatment Study of Mucopolysaccharidosis Type IIIB

Clinical Trial Summary

The study's primary objectives are to evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of AX 250 on cognitive function in patients with MPS IIIB as assessed by the Development Quotient.

See also
Status	Clinical Trial	Phase
Completed	`NCT02493998` - A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Active, not recruiting	`NCT03227042` - A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Completed	`NCT02037880` - Natural History Studies of Mucopolysaccharidosis III	N/A
Active, not recruiting	`NCT03784287` - A Treatment Extension Study of Mucopolysaccharidosis Type IIIB	Phase 2

See also

Status

Clinical Trial

Phase

Completed

NCT02493998 - A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Active, not recruiting

NCT03227042 - A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Completed

NCT02037880 - Natural History Studies of Mucopolysaccharidosis III

N/A

Active, not recruiting

NCT03784287 - A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Phase 2

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	NCT02754076
Study type	Interventional
Source	Allievex Corporation
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	April 2016
Completion date	July 31, 2020

A Treatment Study of Mucopolysaccharidosis Type IIIB — MPS IIIB

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms