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Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

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ClinicalTrials.gov Identifier: NCT02156674
Recruitment Status : Terminated (Slow accrual)
First Posted : June 5, 2014
Results First Posted : December 23, 2020
Last Update Posted : December 23, 2020
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

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.

Condition or disease Intervention/treatment Phase
Maroteaux-Lamy Syndrome Drug: Naglazyme® Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
Actual Study Start Date : January 26, 2016
Actual Primary Completion Date : December 1, 2019
Actual Study Completion Date : December 1, 2019


Arm Intervention/treatment
Experimental: Naglazyme®
weekly Naglazyme® infusion for 2 years
Drug: Naglazyme®
1 mg per kg of body weight administered once weekly as an intravenous infusion




Primary Outcome Measures :
  1. Change in Urinary Glycosaminoglycan (GAG) Excretion [ Time Frame: Baseline and 2 years ]
    Change in urinary glycosaminoglycan (GAG) excretion from baseline to 2 years

  2. Change in Distance Traveled [ Time Frame: Baseline and 2 years ]
    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

  3. Change in Neurocognitive Ability [ Time Frame: Baseline and 2 years ]
    Subjects will undergo full neuropsychological testing annually and abbreviated testing for attention and adaptive skills at the six-month interval visits


Secondary Outcome Measures :
  1. Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy [ Time Frame: 6 months ]

    Development of antibodies including neutralizing antibodies with weekly Naglazyme therapy and the impact of these antibodies on cardiorespiratory and skeletal parameters as measured by the 6--minute walk test and standard tests of range of motion and mobility, and the change in neurocognitive ability.

    Anti-rhASB antibodies in patient's sera were tested in the Biomarin Laboratory using an in-house test of ELISA.


  2. Number of Participants With Development of Neutralizing Antibodies to Naglazyme Therapy [ Time Frame: 2 years ]

    Development of antibodies including neutralizing antibodies with weekly Naglazyme therapy and the impact of these antibodies on cardiorespiratory and skeletal parameters as measured by the 6--minute walk test and standard tests of range of motion and mobility, and the change in neurocognitive ability.

    Anti-rhASB antibodies in patient's sera were tested in the Biomarin Laboratory using an in-house test of ELISA.




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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02156674


Locations
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United States, Minnesota
University of Minnesota Medical Center, Fairview
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
BioMarin Pharmaceutical
Investigators
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Principal Investigator: Elizabeth Braulin, M.D. University of Minnesota
  Study Documents (Full-Text)

Documents provided by Masonic Cancer Center, University of Minnesota:
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT02156674    
Other Study ID Numbers: 2014LS014
MT2014-08R ( Other Identifier: University of Minnesota Blood and Marrow Transplant Program )
First Posted: June 5, 2014    Key Record Dates
Results First Posted: December 23, 2020
Last Update Posted: December 23, 2020
Last Verified: December 2020
Keywords provided by Masonic Cancer Center, University of Minnesota:
Maroteaux-Lamy Syndrome
mucopolysaccharide VI
MPS VI
Additional relevant MeSH terms:
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Mucopolysaccharidosis VI
Syndrome
Disease
Pathologic Processes
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases