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Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

NCT ID: NCT02156674

Last Updated: 2020-12-23

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-01-26

Study Completion Date

2019-12-01

Brief Summary

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This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.

Detailed Description

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Conditions

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Maroteaux-Lamy Syndrome

Keywords

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Maroteaux-Lamy Syndrome mucopolysaccharide VI MPS VI

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Naglazyme®

weekly Naglazyme® infusion for 2 years

Group Type EXPERIMENTAL

Naglazyme®

Intervention Type DRUG

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

Interventions

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Naglazyme®

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

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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
* Age \> 2 years
* \>10% engrafted based on most recent testing
* Willing to commit to traveling to the University of Minnesota every 6 months
* Written informed consent with parent/guardian consent for children \< 18 years of age or persons unable to consent with minor assent if appropriate

Exclusion Criteria

* History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen
* Pregnant or breastfeeding
* Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study
Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioMarin Pharmaceutical

INDUSTRY

Sponsor Role collaborator

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Elizabeth Braulin, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Locations

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University of Minnesota Medical Center, Fairview

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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MT2014-08R

Identifier Type: OTHER

Identifier Source: secondary_id

2014LS014

Identifier Type: -

Identifier Source: org_study_id