Trials Sponsors Conditions Drugs Pricing

Conditions

Maroteaux-Lamy Syndrome

Keywords

Maroteaux-Lamy Syndromemucopolysaccharide VIMPS VI

Outcome Measures

Primary Outcomes (3)

Change in Urinary Glycosaminoglycan (GAG) Excretion
Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years
Baseline and 2 years
Change in Distance Traveled
Change in distance traveled in 6 minute walk and standard tests of range of motion and mobility. Improvement in distance walked from the start to study to end of the study is reported
Baseline and 2 years
Change in Neurocognitive Ability
Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits
Baseline and 2 years

Secondary Outcomes (2)

Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
6 months
Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy
2 years

Study Arms (1)

Naglazyme®

EXPERIMENTAL

weekly Naglazyme® infusion for 2 years

Drug: Naglazyme®

Interventions

Naglazyme®Drug

1 mg per kg of body weight administered once weekly as an intravenous infusion

Naglazyme®

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age3 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant \>2 years previously
Persons currently receiving Naglazyme may be accepted into the study

Sponsors & Collaborators

Masonic Cancer Center, University of Minnesota
Otherlead
BioMarin Pharmaceutical
Industrycollaborator

Study Sites (1)

University of Minnesota Medical Center, Fairview

Minneapolis, Minnesota, 55455, United States

MeSH Terms

Conditions

Mucopolysaccharidosis VI

Interventions

galsulfase

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title: Elizabeth Braunlin MD, PhD
Organization: Masonic Cancer Center, University of Minnesota

Study Officials

Elizabeth Braulin, M.D.
University of Minnesota
PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee: Yes

Enrollment

1 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: not applicable
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

June 3, 2014

First Posted

June 5, 2014

Study Start

January 26, 2016

Actual (reported)

Primary Completion

Study Stopped

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (2)

Study Arms (1)

Naglazyme®

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (2)

Study Arms (1)

Naglazyme®

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations