Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

NCT02156674 | Terminated Results DMC

• 2 other identifiers: 2014LS014MT2014-08R
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Maroteaux-Lamy Syndrome

Keywords

Maroteaux-Lamy Syndrome; mucopolysaccharide VI; MPS 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®

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead
• BioMarin Pharmaceutical: collaborator

Study Sites (1)

University of Minnesota Medical Center, Fairview

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis VI

Interventions

galsulfase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

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

braun002@umn.edu

612 626 2755

Study Officials

• Elizabeth Braulin, M.D.

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

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
• Primary Completion: December 1, 2019
• Study Completion: December 1, 2019
• Last Updated: December 23, 2020
• Results First Posted: December 23, 2020

Record last verified: 2020-12

Locations

US (1)