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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/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
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)
Secondary ID [1] 0 0
110-502
Universal Trial Number (UTN)
Trial acronym
MOR-AUS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis IVA (Morquio A Syndrome) 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety Analysis
Timepoint [1] 0 0
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.
Secondary outcome [1] 0 0
Efficacy Analysis
Timepoint [1] 0 0
Baseline, Week 25, Week 49 or Early Termination Visit
Secondary outcome [2] 0 0
Efficacy Analysis (recommended)
Timepoint [2] 0 0
by Week 24, Week 52 and Early Termination Visit during Extension Phase

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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/07/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2016
Sample size
Target
Accrual to date
Final
13
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Lady Cilento Children's Hospital (previous: Royal Children's Hospital) - Brisbane
Recruitment hospital [3] 0 0
Murdoch Childrens Research Institute and Royal Children's Hospital - Melbourne
Recruitment hospital [4] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment hospital [5] 0 0
Pain and Anaesthesia Research Clinic, Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4101 (4029) - Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Melbourne
Recruitment postcode(s) [4] 0 0
6008 - Perth
Recruitment postcode(s) [5] 0 0
5OOO - Adelaide

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

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
Trial website
https://clinicaltrials.gov/ct2/show/NCT01966029
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Tristan Zhang, MD
Address 0 0
BioMarin Pharmaceutical
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries