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Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01697319
Recruitment Status : Terminated
First Posted : October 2, 2012
Results First Posted : January 12, 2016
Last Update Posted : January 12, 2016
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE September 17, 2012
First Posted Date  ICMJE October 2, 2012
Results First Submitted Date  ICMJE October 12, 2015
Results First Posted Date  ICMJE January 12, 2016
Last Update Posted Date January 12, 2016
Study Start Date  ICMJE August 2012
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 8, 2015)
  • 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.
Original Primary Outcome Measures  ICMJE
 (submitted: September 27, 2012)
Efficacy will be determined/summarized by percent changes of the domain scores for upper extremity function, dexterity, mobility, pain, and self-care and functional ability testing from baseline. [ Time Frame: Up to 48 weeks ]
  • Upper extremity function will be tested using the Grip-Pinch Test.
  • Dexterity will be tested using the Functional Dexterity Test.
  • Mobility will be tested using the 25 Foot Walk test
  • Pain will be determined by the Brief Pain Inventory-Short Form questionnaire or the Adolescent Pediatric Pain Tool
  • Functional/self care abilities will be determined using Pediatric Outcomes Data Collection Instrument (PODCI) or the SF-36 questionnaire.
Efficacy will be measured at the following timepoints: Baseline, Week 12, Week 24, Week 36,and Week 48/ ETV
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 8, 2015)
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.
Original Secondary Outcome Measures  ICMJE
 (submitted: September 27, 2012)
  • Change in respiratory function using a summarized analysis of the percentage change in FET, FIVC, FVC, FEV1, MVV and optional TLC values from the Baseline visit. [ Time Frame: Up to 48 weeks ]
    Timepoints: Baseline, Week 24, and Week 48
  • Change in plasma and urinary KS over time as determined by descriptive statistics. [ Time Frame: Up to 48 weeks ]
    Timepoints: Baseline, Week 2, Week 4, Week 6, Week 12, Week 24, Week 36, Week 48/ ETV
  • Assessment of effect on sleep apnea results as calculated using the Apnea-hypopnea index(AHI). [ Time Frame: Up to 48 weeks ]
    A subset of approximately 5 patients at selected sites who have abnormal overnight pulse oximetry readings and who provide additional informed consent will be assessed for sleep apnea.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: September 27, 2012)
Descriptive summary of clinical safety assessments [ Time Frame: Continuously for up to 48 weeks or more ]
Incidence of AEs and changes in neurologic examinations, vital signs, ECHOs, ECGs, cervical spine radiographs, immunogenicity tests, clinical laboratory tests, and concomitant medications.
Descriptive Information
Brief Title  ICMJE Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Official Title  ICMJE 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
Brief Summary The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.
Detailed Description

Effect is defined by the following key domains:

  • Upper extremity function and dexterity
  • Mobility
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
Intervention  ICMJE 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:
  • N-acetylgalactosamine-6-sulfatase
  • N-acetylgalactosamine-6-sulfate
  • sulfatase
  • galactose-6-sulfatase
  • enzyme replacement therapy
  • ERT
  • elosulfase alfa
Study Arms  ICMJE 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.
Intervention: Drug: BMN 110
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: December 8, 2015)
Original Estimated Enrollment  ICMJE
 (submitted: September 27, 2012)
Actual Study Completion Date  ICMJE October 2014
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany,   United Kingdom,   United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT01697319
Other Study ID Numbers  ICMJE MOR-006
2011-005703-33 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Celeste Decker, M.D. BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date December 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP