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Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01415427
First received: August 8, 2011
Last updated: April 13, 2017
Last verified: April 2017
  Purpose
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).

Condition Intervention Phase
Mucopolysaccharidosis IV A Morquio A Syndrome MPS IVA Drug: BMN 110 - Weekly Drug: BMN 110 - Every Other Week Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official 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)

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Change From Baseline in 6-minute Walk (6MW) Test - ITT [ Time Frame: Baseline to week 168 ]
    Efficacy was assessed by changes from baseline in 6-minute walk test

  • Change From Baseline in 6-minute Walk (6MW) Test - MPP [ Time Frame: Baseline to week 168 ]
    Efficacy was assessed by changes from baseline in 6-minute walk test


Secondary Outcome Measures:
  • Change From Baseline in 3-minute Stair Climb Test - ITT [ Time Frame: Baseline to week 168 ]
    Efficacy was assessed by changes from baseline in 3-minute stair climb test.

  • Change From Baseline in 3-minute Stair Climb Test - MPP [ Time Frame: Baseline to week 168 ]
    Efficacy was assessed by changes from baseline in 3-minute stair climb test.

  • Change From Baseline in Urine Keratan Sulfate - ITT [ Time Frame: Baseline to week 168 ]
    Efficacy was assessed by changes from baseline in urine keratan sulfate (normalized to urine creatinine.)

  • Change From Baseline in Urine Keratan Sulfate - MPP [ Time Frame: Baseline to week 168 ]
    Efficacy was assessed by changes from baseline in urine keratan sulfate (normalized to urine creatinine.)


Enrollment: 173
Study Start Date: July 2011
Study Completion Date: June 16, 2016
Primary Completion Date: June 16, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BMN 110 Weekly
BMN 110 Weekly: In Part 1, patients will receive an intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
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.

Other Names:
  • N-acetylgalactosamine-6-sulfatase
  • N-acetylgalactosamine-6-sulfate sulfatase
  • galactose-6-sulfatase
  • GALNS
  • enzyme replacement therapy
  • ERT
Experimental: BMN 110 Every Other Week
BMN 110 Every Other Week: In Part 1, patients will receive an intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and will receive infusions of placebo on alternating weeks.
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.

Other Names:
  • N-acetylgalactosamine-6-sulfatase
  • N-acetylgalactosamine-6-sulfate sulfatase
  • galactose-6-sulfatase
  • GALNS
  • enzyme replacement therapy
  • ERT

Detailed Description:

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
  Eligibility

Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01415427

  Show 43 Study Locations
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Debra Lounsbury BioMarin Pharmaceutical
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01415427     History of Changes
Other Study ID Numbers: MOR-005
Study First Received: August 8, 2011
Results First Received: April 13, 2017
Last Updated: April 13, 2017

Keywords provided by BioMarin Pharmaceutical:
Mucopolysaccharidosis IV type A
MPS IV Type A
Mucopolysaccharidosis IVA
MPS IVA
Morquio A Syndrome
Lysosomal Storage Disorder
LSD
N-acetylgalactosamine-6-sulfatase
N-acetylgalactosamine-6-sulfate sulfatase
galactose-6-sulfatase
GALNS
enzyme replacement therapy
ERT

Additional relevant MeSH terms:
Syndrome
Mucopolysaccharidoses
Mucopolysaccharidosis IV
Osteochondrodysplasias
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases

ClinicalTrials.gov processed this record on June 23, 2017