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

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	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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:

Genetics Home Reference related topics: mucopolysaccharidosis type IV Schindler disease

Drug Information available for: Sulfate ion

U.S. FDA Resources

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

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.

Secondary Outcome Measures:

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.

Estimated Enrollment:	162
Study Start Date:	July 2011
Estimated Study Completion Date:	December 2017
Estimated Primary Completion Date:	May 2017 (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 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. 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 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. Other Names: N-acetylgalactosamine-6-sulfatase N-acetylgalactosamine-6-sulfate sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT

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

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.

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

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.

Other Names:

N-acetylgalactosamine-6-sulfatase
N-acetylgalactosamine-6-sulfate sulfatase
galactose-6-sulfatase
GALNS
enzyme replacement therapy
ERT

Eligibility

Ages Eligible for Study:	5 Years and older
Genders Eligible for Study:	Both
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

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

Show 33 Study Locations

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

Study Director:

Debra Lounsbury, R.N, M.S.

BioMarin Pharmaceutical

More Information

No publications provided

Responsible Party:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT01415427 History of Changes
Other Study ID Numbers:	MOR-005
Study First Received:	August 8, 2011
Last Updated:	September 18, 2012
Health Authority:	United States: Food and Drug Administration

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:

Mucopolysaccharidoses
Mucopolysaccharidosis IV
Osteochondrodysplasias
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 17, 2013