Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation



Status:Terminated
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:5 - Any
Updated:4/21/2016
Start Date:August 2012
End Date:October 2014

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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

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.

Effect is defined by the following key domains:

- Upper extremity function and dexterity

- Mobility

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.
We found this trial at
3
sites
225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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747 52nd St
Oakland, California 94609
(510) 428-3000
Children's Hospital and Research Center Oakland For nearly 100 years, Children's Hospital & Research Center...
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Hamburg,
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