Clinical trial NCT01697319: Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Official title: A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Mucopolysaccharidosis IVA
Morquio A Syndrome
MPS IVA
Keywords: Mucopolysaccharidosis IVA Type A
MPS IVA 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
MOR-006
CPET
Limited ambulation
Grip/ Pinch
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2012-10-02
Enrollment: 16
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.

Detailed description: Effect is defined by the following key domains:

* Upper extremity function and dexterity
* Mobility

Eligibility criteria: Inclusion Criteria:

* Is willing and able to provide written, signed informed consent (or their legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure. Patients who do not meet country and local age requirements for informed consent must be willing and able to provide written assent after the nature of the study has been explained and prior to performance of any research-related procedure.
* Has documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA.
* Is ≥ 5 years of age.
* If sexually active, is willing to use an acceptable method of contraception while participating in the study.
* Females of childbearing potential must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
* Is willing and able to perform all study procedures as physically possible.

Exclusion Criteria:

* Is able to walk farther than a specified distance as assessed by the 6MWT.
* Has previous hematopoietic stem cell transplant (HSCT).
* Has received previous treatment with BMN 110.
* Has a known hypersensitivity to any of the components of BMN 110.
* Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the first 24 weeks of the study.
* Has used any other investigational product or investigational medical device within 30 days prior to the Screening Visit or requires any investigational agent prior to completion of all scheduled study assessments.
* Is pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study.
* Has a concurrent disease or condition, including but not limited to symptomatic cervical spine instability or severe cardiac disease or complete paralysis due to a spinal cord injury (defined as an inability to move arms and legs), that would interfere with study participation or safety 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.

Primary outcomes:
measure: Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
timeFrame: Up to 96 weeks
measure: Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
timeFrame: Up to 96 weeks
measure: Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)
timeFrame: Up to 96 weeks
Secondary outcomes:
measure: Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)
timeFrame: Up to 96 weeks
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital & Research Center Oakland
state: California
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Hamburg
country: Germany
facility: Universitätsklinikum Hamburg
city: Mainz
country: Germany
facility: University Medical Center Mainz, Center of Pediatric and Adolescent Medicine Villa Metabolica
city: Birmingham
country: United Kingdom
facility: NIHR/Wellcome Trust Birmingham CRF, Queen Elizabeth Hospital
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospitals NHS Foundation Trust
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
Study officials:
affiliation: BioMarin Pharmaceutical
name: Celeste Decker, M.D.
role: STUDY_DIRECTOR
References: