Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
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ClinicalTrials.gov Identifier: NCT01697319 |
Recruitment Status :
Terminated
First Posted : October 2, 2012
Results First Posted : January 12, 2016
Last Update Posted : January 12, 2016
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Condition or disease | Intervention/treatment | Phase |
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Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA | Drug: BMN 110 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 16 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation |
Study Start Date : | August 2012 |
Actual Primary Completion Date : | October 2014 |
Actual Study Completion Date : | October 2014 |

Arm | Intervention/treatment |
---|---|
Experimental: BMN 110 at 2.0 mg/kg/week
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 144 weeks.
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Drug: BMN 110
Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.
Other Names:
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- Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) [ Time Frame: Up to 96 weeks ]FDT assesses the ability to use the hand in daily tasks. The test involves turning 16 wooden pegs over as quickly as possible on a hardwood pegboard with one hand requiring a three-jaw chuck prehension pattern between the fingers and thumb within a two-minute time limit. Hand function is evaluated by how fast a patient can turn over pegs in the given time limit, i.e. speed (number of pegs/minute).
- Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT) [ Time Frame: Up to 96 weeks ]A grip-strength dynamometer and a pinch meter were used to measure grip strength and pinch strength. The results report change from baseline in strength for dominant and non-dominant hand in a forearm and wrist supported position.
- Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT) [ Time Frame: Up to 96 weeks ]The timed 25-Foot Walk Test (25FWT) is an assessment of mobility and performance of leg function. The patient was instructed to walk a marked 25-foot course as quickly as possible in a time limit of 3 minutes and immediately walk back the same distance when reaching one end.The patient is allowed to use any ambulation method to move. The outcome measures the speed (feet / min) of moving.
- Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS) [ Time Frame: Up to 96 weeks ]Urinary keratan sulfate and urinary creatinine were measured through quantitative analysis. uKS is normalized to creatinine.

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Ages Eligible for Study: | 5 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Is willing and able to provide written, signed informed consent (or their legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure. Patients who do not meet country and local age requirements for informed consent must be willing and able to provide written assent after the nature of the study has been explained and prior to performance of any research-related procedure.
- Has 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.
- Is ≥ 5 years of age.
- If sexually active, is willing to use an acceptable method of contraception while participating in the study.
- Females of childbearing potential must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
- Is willing and able to perform all study procedures as physically possible.
Exclusion Criteria:
- Is able to walk farther than a specified distance as assessed by the 6MWT.
- Has previous hematopoietic stem cell transplant (HSCT).
- Has received previous treatment with BMN 110.
- Has a known hypersensitivity to any of the components of BMN 110.
- Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the first 24 weeks of the study.
- Has used any other investigational product or investigational medical device within 30 days prior to the Screening Visit or requires any investigational agent prior to completion of all scheduled study assessments.
- Is pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study.
- Has a concurrent disease or condition, including but not limited to symptomatic cervical spine instability or severe cardiac disease or complete paralysis due to a spinal cord injury (defined as an inability to move arms and legs), that would interfere with study participation or safety as determined by the Investigator.
- Has 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.

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): NCT01697319
United States, California | |
Children's Hospital & Research Center Oakland | |
Oakland, California, United States | |
United States, Illinois | |
Ann & Robert H. Lurie Children's Hospital of Chicago | |
Chicago, Illinois, United States | |
Germany | |
Universitätsklinikum Hamburg | |
Hamburg, Germany | |
University Medical Center Mainz, Center of Pediatric and Adolescent Medicine Villa Metabolica | |
Mainz, Germany | |
United Kingdom | |
NIHR/Wellcome Trust Birmingham CRF, Queen Elizabeth Hospital | |
Birmingham, United Kingdom | |
Central Manchester University Hospitals NHS Foundation Trust | |
Manchester, United Kingdom | |
Salford Royal NHS Foundation Trust | |
Salford, United Kingdom |
Study Director: | Celeste Decker, M.D. | BioMarin Pharmaceutical |
Responsible Party: | BioMarin Pharmaceutical |
ClinicalTrials.gov Identifier: | NCT01697319 |
Other Study ID Numbers: |
MOR-006 2011-005703-33 ( EudraCT Number ) |
First Posted: | October 2, 2012 Key Record Dates |
Results First Posted: | January 12, 2016 |
Last Update Posted: | January 12, 2016 |
Last Verified: | December 2015 |
Morquio A Syndrome Mucopolysaccharidosis IVA Type A MPS IVA Type A Mucopolysaccharidosis IVA MPS IVA Lysosomal Storage Disorder LSD N-acetylgalactosamine-6-sulfatase N-acetylgalactosamine-6-sulfate |
sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT MOR-006 CPET Limited ambulation Grip/ Pinch |
Mucopolysaccharidoses Mucopolysaccharidosis IV Syndrome Disease Pathologic Processes Carbohydrate Metabolism, Inborn Errors |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |