Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

See also
Status	Clinical Trial	Phase
Completed	`NCT00299000` - A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI	Phase 4
Terminated	`NCT00668564` - Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism	Phase 2
Completed	`NCT01043640` - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Phase 2

See also

Status

Clinical Trial

Phase

Completed

NCT00299000 - A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Phase 4

Terminated

NCT00668564 - Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Phase 2

Completed

NCT01043640 - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

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	NCT02156674
Study type	Interventional
Source	Masonic Cancer Center, University of Minnesota
Contact
Status	Terminated
Phase	N/A
Start date	January 26, 2016
Completion date	December 1, 2019

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms