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)

Trial Source

There is no location for this trial

Location data is sourced from multiple external providers and UKCTG is not responsible for and cannot guarantee the accuracy of data.

Health Conditions
  • Mucopolysaccharidosis IV A
  • Morquio A Syndrome
Unfortunately contact details are not available for this trial.
Primary Contact Details
Not Recruiting
Recruitment Status
Primary Trial ID Number
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).
Research Details
  • This is a multi-center, multinational, extension study to evaluate 2 dose regimens of BMN 110 treatment in patients with MPS IVA who completed MOR-004. The last study visit assessments for MOR-004 will constitute Baseline for this study. The first study drug dose of this protocol will occur on Week 0 of MOR-005, which is the same as the last visit (Week 24) of MOR-004. Initially, the study will be double-blind with patients previously randomized to BMN 110 in MOR-004 remaining on their assigned BMN 110 dose regimen (qw or qow dosing). The MOR-004 placebo patients will be re-randomized (1:1 ratio) to one of the 2 BMN 110 dose regimen groups: 2.0 mg/kg/qw or 2.0 mg/kg/qow. There will be two study parts: - Part 1 - randomized double-blind until the optimal BMN 110 dose regimen has been determined, based on the final primary efficacy analysis from MOR-004 - Part 2 - open-label BMN 110 treatment with the single optimal dose regimen
Phase 3
Study Design
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Study Type
Drug : BMN 110 - Weekly, Drug : BMN 110 - Every Other Week

Study Arm Groups : BMN 110 Weekly, BMN 110 Every Other Week

Intervention Type
See Interventions above
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
Sorry, this information is not available
Result Reports
This is available on the Clinicaltrials.gov website
Age Range
5 Years - N/A
Who Can Participate
Number of Participants
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
This is in the inclusion criteria above
Trial Location(s)
Receptos Study Site 967
B15 2TH
West Midlands
B4 6NH
Ulster Hospital
Trial Contact(s)
Primary Trial Contact
Sorry, this information is not available
Other Trial Contacts
Sorry, this information is not available
Countries Recruiting
Argentina, Brazil, Canada, Colombia, Denmark, France, Germany, Italy, Japan, Korea, Republic of, Netherlands, Norway, Portugal, Qatar, Saudi Arabia, Spain, Taiwan, United Kingdom, United States
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)
EudraCT Number
Not available for this trial
    Sorry, this information is not available
Other Study ID Numbers
BioMarin Pharmaceutical
Key Dates

Recruitment Start Date

Jul 2011

Recruitment End Date

May 2017

Trial Start Date
Date Not Available
Trial End Date
Date Not Available
Date Assigned

08 Aug 2011

Last Updated

22 Apr 2014