Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type

See also
	Status	Clinical Trial	Phase
	Enrolling by invitation	`NCT04360265` - A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102	Phase 3
	Recruiting	`NCT05523206` - A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

See also

Status

Clinical Trial

Phase

Enrolling by invitation

NCT04360265 - A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Phase 3

Recruiting

NCT05523206 - A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

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	NCT06095388
Study type	Interventional
Source	JCR Pharmaceuticals Co., Ltd.
Contact	JCR Pharmaceuticals Co., Ltd.
Phone	+81-(0)797-32-8582
Email	clinical_development@jp.jcrpharm.com
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	October 4, 2023
Completion date	October 31, 2029

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms