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Research Question
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This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0
mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis
IVA (Morquio A Syndrome).
(from ClinicalTrials.gov)
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Ethics Approval
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Sorry, not currently available
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Study Design
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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Design Type
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Sorry, not currently available
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Design Details
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Sorry, not currently available
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Health Condition(s) or Problem
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Mucopolysaccharidosis IV A; Morquio A Syndrome; MPS IVA
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Participants Inclusion Criteria
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Inclusion Criteria:
- Must have completed MOR-004
- Is 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 authorize representative, after the nature of the study has been explained,
and prior to performance of research-related procedures.
- If sexually active, must be willing to use an acceptable method of contraception
while participating in the study.
- If female, of childbearing potential, must have a negative pregnancy test at Baseline
and be willing to have additional pregnancy tests done during the study.
Exclusion Criteria:
- Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or
partner) at any time during the study.
- Has used any investigational product (other than BMN 110 in MOR-004), 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.
- Was enrolled in a previous BMN 110 study, other than MOR-004.
- Has a concurrent disease or condition, including but not limited to, symptomatic
cervical spine instability, clinically significant spinal cord compression, or severe
cardiac disease that would interfere with study participation, or pose a safety risk,
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.
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Participants Exclusion Criteria
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Inclusion Criteria:
- Must have completed MOR-004
- Is 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 authorize representative, after the nature of the study has been explained,
and prior to performance of research-related procedures.
- If sexually active, must be willing to use an acceptable method of contraception
while participating in the study.
- If female, of childbearing potential, must have a negative pregnancy test at Baseline
and be willing to have additional pregnancy tests done during the study.
Exclusion Criteria:
- Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or
partner) at any time during the study.
- Has used any investigational product (other than BMN 110 in MOR-004), 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.
- Was enrolled in a previous BMN 110 study, other than MOR-004.
- Has a concurrent disease or condition, including but not limited to, symptomatic
cervical spine instability, clinically significant spinal cord compression, or severe
cardiac disease that would interfere with study participation, or pose a safety risk,
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.
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Participant Sex
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Sorry, not currently available
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Participant Age Range
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Sorry, not currently available
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Participant Type
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Sorry, not currently available
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Target Sample Size
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162
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Date of First Enrolment
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Sorry, not currently available
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Date of Recruitment End
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Sorry, not currently available
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Date of End of Follow-up
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Sorry, not currently available
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Trial End Date
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Sorry, not currently available
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Recruitment Status
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Not Recruiting
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Overall Trial Status
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Sorry, not currently available
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Countries of Recruitment
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United States; Argentina; Brazil; Canada; Colombia; Denmark; France; Germany; Italy; Japan; Korea, Republic of; Netherlands; Norway; Portugal; Qatar; Saudi Arabia; Taiwan; United Kingdom
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Nation
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England
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Location
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Birmingham, London, Manchester
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Interventions
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Drug; BMN 110; In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of either 2.0 mg/kg/qw or 2.0 mg/kg/qow. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks. In Part 2, patients will receive 2.0 mg/kg of BMN 110 either every week or every other week, with no placebo.; BMN 110 Weekly; BMN 110 Every Other Week; N-acetylgalactosamine-6-sulfatase; N-acetylgalactosamine-6-sulfate sulfatase; galactose-6-sulfatase; GALNS; enzyme replacement therapy; ERT
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Primary Outcome Measures
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Primary Long-Term Safety/Efficacy Evaluation
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Secondary Outcome Measures
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Long-Term evaluation of changes in biochemical markers of inflammation and bone and cartilage metabolism; Approximately 240 weeks; Yes; To evaluate the long-term effect of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow on changes in biochemical markers of inflammation and bone and cartilage metabolism, in patients with MPS IVA.
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Website
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Sorry, not currently available
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