Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Not Recruiting
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Health Conditions
  • Mucopolysaccharidosis IV A
  • Morquio A Syndrome
  • MPS IVA
Not Recruiting
Recruitment Status
NCT01415427
Primary Trial ID Number
Summary
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).
Primary Outcome Measures
  • Primary Long-Term Safety/Efficacy Evaluation; Approximately 240 weeks
Secondary Outcome Measures
  • Long-Term evaluation of changes in biochemical markers of inflammation and bone and cartilage metabolism; Approximately 240 weeks
Research Question
  • 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).
Design Type
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Ethics Approval
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Publications
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Countries of Recruitment
United States; Argentina; Brazil; Canada; Colombia; Denmark; France; Germany; Italy; Japan; Korea, Republic of; Netherlands; Norway; Portugal; Qatar; Saudi Arabia; Spain; Taiwan; United Kingdom
Participant Sex
Both
Participant Age Range
5 Years to N/A
Participant Type
Sorry, this information is not available
Trial Sample Size
173
Participant Inclusion Criteria
  • 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.
Participant Exclusion Criteria
  • 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.
Interventions
Drug; BMN 110 - Weekly; In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week. In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo.; [BMN 110 Weekly]; Drug; BMN 110 - Every Other Week; In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. 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 every week, with no placebo.; [BMN 110 Every Other Week]
Design Details
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Study Design
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Results Reporting
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Acronym
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Scientific Title
A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Secondary Trial Identifying Number
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Website
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Study Funded By
BioMarin Pharmaceutical
Funder Type
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Study Sponsored By
BioMarin Pharmaceutical
Study Also Sponsored By
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Primary Sponsor Type
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Secondary Sponsor Type
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Key Dates

Date of First Enrollment
Date Not Available
Recruitment End Date
Date Not Available
Trial End Date
Date Not Available
Date added to Registry

08 Aug 2011

Last Updated

22 Apr 2014

Date Record Refreshed on UKCTG

31 Jul 2015