Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation (NCT01697319) | Clinical Trial Compass
TerminatedPhase 2
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
United States, Germany, United Kingdom16 participantsStarted 2012-08
Plain-language 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.
Who can participate
Age range5 Years
SexALL
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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 an…
What they're measuring
1
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
Timeframe: Up to 96 weeks
2
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
Timeframe: Up to 96 weeks
3
Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)