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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01415427
First received: August 8, 2011
Last updated: April 22, 2014
Last verified: April 2014

August 8, 2011
April 22, 2014
July 2011
May 2017   (final data collection date for primary outcome measure)
Primary Long-Term Safety/Efficacy Evaluation [ Time Frame: Approximately 240 weeks ] [ Designated as safety issue: Yes ]
To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA. Safety results will be determined by numbers and severity of adverse events as well as descriptive statistic analysis of other safety related assessments. Efficacy will be assessed by changes from baseline in 6-minute walk test and 3-minute stair climb test as well as urine keratan sulfate (normalized to urine creatinine.)
Primary Long-Term Safety/Efficacy Evaluation [ Time Frame: Approximately 240 weeks ] [ Designated as safety issue: Yes ]
To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA. Safety results will be determined by numbers and severity of adverse events as well as descriptive statistic analysis of other safety related assessments. Efficacy will be assessed by changes from baseline in 6-minute walk test and 3-minute stair climb test as well as urine KS concentrations.
Complete list of historical versions of study NCT01415427 on ClinicalTrials.gov Archive Site
Long-Term evaluation of changes in biochemical markers of inflammation and bone and cartilage metabolism [ Time Frame: Approximately 240 weeks ] [ Designated as safety issue: 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.
Same as current
Not Provided
Not Provided
 
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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).

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
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Mucopolysaccharidosis IV A
  • Morquio A Syndrome
  • MPS IVA
  • 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
  • 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
  • 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.
    Intervention: Drug: BMN 110 - Weekly
  • 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.
    Intervention: Drug: BMN 110 - Every Other Week
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
173
December 2017
May 2017   (final data collection date for primary outcome measure)

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.
Both
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Argentina,   Brazil,   Canada,   Colombia,   Denmark,   France,   Germany,   Italy,   Japan,   Korea, Republic of,   Netherlands,   Norway,   Portugal,   Qatar,   Saudi Arabia,   Spain,   Taiwan,   United Kingdom
 
NCT01415427
MOR-005
Yes
BioMarin Pharmaceutical
BioMarin Pharmaceutical
Not Provided
Study Director: Matthew Mealiffe, M.D. BioMarin Pharmaceutical
BioMarin Pharmaceutical
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP