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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01966029
Registration number
NCT01966029
Ethics application status
Date submitted
10/09/2013
Date registered
21/10/2013
Date last updated
26/09/2019
Titles & IDs
Public title
BMN 110 Phase 3B in Australian Patients
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Scientific 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)
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Secondary ID [1]
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110-502
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Universal Trial Number (UTN)
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Trial acronym
MOR-AUS
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis IVA (Morquio A Syndrome)
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Condition category
Condition code
Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - BMN 110
Experimental: Treatment - All patients receive treatment with BMN 110
Treatment: Drugs: BMN 110
All pateints receive treatment with BMN 110
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Safety Analysis
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Assessment method [1]
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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.
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Timepoint [1]
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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.
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Secondary outcome [1]
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Efficacy Analysis
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, Week 25, Week 49 or Early Termination Visit
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Secondary outcome [2]
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Efficacy Analysis (recommended)
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Assessment method [2]
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The efficacy assessments as mentioned in Efficacy Analysis 2 above are all recommended in the Extension Phase per the protocol amendment dated 17Mar 2014
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Timepoint [2]
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by Week 24, Week 52 and Early Termination Visit during Extension Phase
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Eligibility
Key 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.
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/07/2013
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/12/2016
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Sample size
Target
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Accrual to date
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Final
13
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
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Recruitment hospital [1]
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Children's Hospital at Westmead - Westmead
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Recruitment hospital [2]
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Lady Cilento Children's Hospital (previous: Royal Children's Hospital) - Brisbane
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Recruitment hospital [3]
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Murdoch Childrens Research Institute and Royal Children's Hospital - Melbourne
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Recruitment hospital [4]
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Princess Margaret Hospital for Children - Perth
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Recruitment hospital [5]
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Pain and Anaesthesia Research Clinic, Royal Adelaide Hospital - Adelaide
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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4101 (4029) - Brisbane
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Recruitment postcode(s) [3]
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3052 - Melbourne
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Recruitment postcode(s) [4]
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6008 - Perth
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Recruitment postcode(s) [5]
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5OOO - Adelaide
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
BioMarin Pharmaceutical
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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
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Trial website
https://clinicaltrials.gov/study/NCT01966029
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Tristan Zhang, MD
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Address
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BioMarin Pharmaceutical
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Fax
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Email
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT01966029
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