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BMN 110 Phase 3B in Australian Patients (MOR-AUS)

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: NCT01966029
Recruitment Status : Completed
First Posted : October 21, 2013
Last Update Posted : September 26, 2019
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:

There is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment option for MPS IVA patients. BMN 110, containing recombinant human GALNS (rhGALNS) developed by BioMarin is expected to reduce the progressive, pathologic accumulation of KS, and improve signs and symptoms of the disease.

The objective of this Phase 3B open label study (110-502) will be to evaluate the safety and tolerability of 2.0 mg/kg/week (qw) of BMN 110 in Australian patients with MPS IVA. In addition, a number of secondary and tertiary efficacy endpoints will also be investigated. The dose and regimen of BMN 110 have been selected on the basis of data from a Phase 1/2 clinical study with BMN 110, nonclinical and in vitro studies with BMN 110, and clinical and nonclinical data from other enzyme replacement therapies.

Extension Phase is included per amendment dated 10Mar 2014: To provide patients enrolled in the Initial Phase access to BMN 110 until commercial product becomes available in Australia and continue to assess long-term safety

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis IVA (Morquio A Syndrome) Drug: BMN 110 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Open-Label, Phase 3B Study to Evaluate the Efficacy and Safety of BMN 110 in Australian Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Study Start Date : July 2013
Actual Primary Completion Date : July 2016
Actual Study Completion Date : December 2016

Arm Intervention/treatment
Experimental: Treatment
All patients receive treatment with BMN 110
Drug: BMN 110
All pateints receive treatment with BMN 110
Other Name: recombinant human N-acetylgalactosamine-6-sulfatase

Primary Outcome Measures :
  1. Safety Analysis [ Time Frame: The study period during which all non-serious AEs and SAEs will be reported begins after informed consent is obtained and through 30 days after the last study visit or 30 days after the last drug infusion, whichever comes first. ]

    The analyses of safety will include all patients who receive any study drug. All safety data will be summarized descriptively. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of AEs will be summarized by System Organ Class (SOC), Preferred Term (PT), relationship to study drug, and severity. Patient listings will be provided for SAEs, deaths, and AEs leading to study drug discontinuation, or study withdrawals. All AE summaries will include only treatment-emergent AEs (TEAEs) reported during the study period (AEs that occur after the first study drug infusion). Infusion-associated AEs will be summarized by SOC, PT and severity.

    The following safety measures will be summarized descriptively: concomitant medications, clinical laboratory tests, vital signs, ECGs, immunogenicity results, analgesic medication use, results from routine physical examinations (including standard neurologic examinations), and cervical spine imaging.

Secondary Outcome Measures :
  1. Efficacy Analysis [ Time Frame: Baseline, Week 25, Week 49 or Early Termination Visit ]

    Efficacy assessments and procedures are the duplicate endurance testing (6MW and 3MSC tests) at Baseline and at week 25 and 49. Samples for urine KS and urine creatinine will be collected at Baseline and every 24 weeks.

    Anthropometric measurements, including standing height, length, sitting height, knee height (as clinically indicated), head circumference and weight, will be taken at Baseline and every 24 weeks.

    Respiratory function tests (RFTs) for assessment of forced expiratory volume for 1 second (FEV1), forced inspiratory vital capacity (FIVC), forced vital capacity (FVC), and maximum voluntary ventilation (MVV) will be performed at Baseline and Weeks 25 and 49. APPT (pain assessment tool), and the PedsQL (Pediatric Quality of Life Inventory) or SF-36® (QOL questionnaire for adult patients) will be done at Baseline, Week 25, and Week 49. The Sleep Apnea Test will be done at Baseline, Week 25 and Week 49, if applicable.

  2. Efficacy Analysis (recommended) [ Time Frame: by Week 24, Week 52 and Early Termination Visit during Extension Phase ]
    The efficacy assessments as mentioned in Efficacy Analysis 2 above are all recommended in the Extension Phase per the protocol amendment dated 17Mar 2014

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Diagnosed with MPS IVA as confirmed by a documented GALNS enzymatic test (GALNS activity in affected range, beta-galactosidase and a second lysosomal sulfatase activity within normal range).
  2. Age 12 months or older.
  3. Willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
  4. If sexually active, must be willing to use an acceptable method of contraception while participating in the study.
  5. If female, and of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy. Childbearing potential will be assessed by the investigator.

Exclusion Criteria:

  1. Previous treatment with BMN 110.
  2. Has known hypersensitivity to any of the components of BMN 110.
  3. Major surgery within 3 months prior to study entry or planned major surgery during the 48-week treatment period.
  4. Prior bone marrow transplant (BMT) or hematopoietic stem cell transplant (HSCT).
  5. Is pregnant or breastfeeding at Baseline, or planning to become pregnant (self orpartner) at any time during the study. Patients who become pregnant during the study will be discontinued from the study.
  6. Has used any investigational product, or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
  7. Has a concurrent disease or condition, including but not limited to, symptomatic cervical spine instability, clinically significant and/or progressive spinal cord compression, or severe cardiac disease that would interfere with study participation, or pose a safety risk, as determined by the Investigator.
  8. 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.

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 identifier (NCT number): NCT01966029

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Australia, New South Wales
Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Lady Cilento Children's Hospital (previous: Royal Children's Hospital)
Brisbane, Queensland, Australia, 4101 (4029)
Australia, Victoria
Murdoch Childrens Research Institute and Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
Australia, Western Australia
Princess Margaret Hospital for Children
Perth, Western Australia, Australia, 6008
Pain and Anaesthesia Research Clinic, Royal Adelaide Hospital
Adelaide, Australia, 5OOO
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Tristan Zhang, MD BioMarin Pharmaceutical
Study Director: Eric He, MD BioMarin Pharmaceutical
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Responsible Party: BioMarin Pharmaceutical Identifier: NCT01966029    
Other Study ID Numbers: 110-502
First Posted: October 21, 2013    Key Record Dates
Last Update Posted: September 26, 2019
Last Verified: September 2019
Additional relevant MeSH terms:
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Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases