Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) - Full Text View

Purpose

This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age, diagnosed with MPS IVA (Morquio A Syndrome) for 52 weeks.

Condition	Intervention	Phase
Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA	Drug: BMN 110	Phase 2

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Resource links provided by NLM:

Genetics Home Reference related topics: mucopolysaccharidosis type IV Schindler disease

Drug Information available for: Sulfate ion

U.S. FDA Resources

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

Descriptive summary of clinical safety assessments [ Time Frame: Up to 52 weeks ] [ Designated as safety issue: Yes ]
Safety will be determined by the following factors:

Number and severity of adverse events in participants. Clinically significant changes in any of the following assessments from Baseline: Vital Signs, echocardiogram, ECG, immunogenicity tests, physical and neurological examinations, standard clinical laboratory tests, concomitant medications, and cervical spine radiography.

Secondary Outcome Measures:

Change in Urinary Keratan Sulfate measures over time [ Time Frame: Baseline, and weeks: 2, 4, 8, 13, 26, 39, and 52/ETV ] [ Designated as safety issue: No ]
To Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce the urinary KS levels in MPS IVA patients less than 5 years.
Change in patient growth over time [ Time Frame: Baseline and Weeks: 13, 26, 39, and 52/ETV ] [ Designated as safety issue: No ]
Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities.

Estimated Enrollment:	15
Study Start Date:	November 2011
Estimated Study Completion Date:	January 2014
Estimated Primary Completion Date:	August 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: BMN 110 Weekly All enrolled patients will receive IV infusions of study drug at a dose of 2.0 mg/kg/wk administered over a period of approximately 4 hours every week for up to 52 weeks.	Drug: BMN 110 Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 52 weeks. Other Names: N-acetylgalactosamine-6-sulfatase N-acetylgalactosamine-6-sulfate sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT

Eligibility

Ages Eligible for Study:	up to 5 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Less than 5 years of age
Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA
Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.

Exclusion Criteria:

Previous hematopoietic stem cell transplant (HSCT).
Previous treatment with BMN 110.
Known hypersensitivity to any of the components of BMN 110.
Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period.
Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator.
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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01515956

Locations

United States, California

Oakland, California, United States
United States, New York

Manhasset, New York, United States
Italy

Monza, Italy
United Kingdom

Central Manchester, United Kingdom

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

Study Director:

Terence Eagleton, MB BS, BSc.

BioMarin Pharmaceutical

More Information

No publications provided

Responsible Party:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT01515956 History of Changes
Other Study ID Numbers:	MOR-007
Study First Received:	December 22, 2011
Last Updated:	August 8, 2012
Health Authority:	United States: Food and Drug Administration United Kingdom: Medicines and Healthcare Products Regulatory Agency United Kingdom: National Institute for Health Research United Kingdom: Research Ethics Committee Italy: The Italian Medicines Agency

Keywords provided by BioMarin Pharmaceutical:

Mucopolysaccharidosis IVA type A
Mucopolysaccharidosis IVA
MPS IVA Type A
MPS IVA
Morquio A Syndrome
Lysosomal Storage Disorder
LSD

N-acetylgalactosamine-6-sulfatase
N-acetylgalactosamine-6-sulfate
sulfatase
galactose-6-sulfatase
GALNS
enzyme replacement therapy
ERT

Additional relevant MeSH terms:

Mucopolysaccharidoses
Mucopolysaccharidosis IV
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn

Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on June 18, 2013