Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (NCT01609062) | Clinical Trial Compass
TerminatedPhase 2
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
United States, Canada25 participantsStarted 2012-04
Plain-language summary
The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks. Secondary objectives were to investigate the effect of the two doses on exercise capacity for up to 196 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 was assessed.
Who can participate
Age range7 Years
SexALL
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Inclusion Criteria:
* Is willing and able to provide written, signed informed consent (or patient's legally authorized representative) after the nature of the study has been explained and prior to performance of any research- related procedure. Also, patients who do not meet country and local age requirements for informed consent must be willing and able to provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to performance of any research-related procedure.
* Has documented clinical diagnosis of Morquio A Syndrome (MPS IVA) based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte N-acetylgalactosamine-6-sulfatase (GALNS) enzyme activity or genetic testing confirming diagnosis of MPS IVA.
* Is at least 7 years of age
* Is able to walk ≥ 200 meters as assessed by the 6-minute Walk Test (6MWT)
* If sexually active, is 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 the Screening Visit and be willing to have additional pregnancy tests during the study
* Is willing and able to perform all study procedures, including cardiopulmonary exercise testing (CPET)
Exclusion Criteria:
* Inability to perform an exercise test due to limited mobility
* Body weight greater than 95 kg at Screening
* Severe, untreated sleep apnea a…
What they're measuring
1
Safety Evaluation
Timeframe: Entire Study Period, up to 192 weeks or ETV (early termination visit)