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Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | Clinical Trials | Clareo Health

TERMINATEDPHASE2

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01609062•BioMarin Pharmaceutical

View on ClinicalTrials.gov

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.

Eligibility

Age

7 - No limit years

Sex

ALL

Healthy Volunteers

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 as measured during Screening with a home sleep testing device
•Patients with a history of, or current condition of sleep apnea or sleep disordered breathing under adequate treatment may be enrolled if approved by the medical monitor.
•Requirement for supplemental oxygen
•Use of ventilator assistance in the 3 months prior to study entry
•Use of positive airway pressure (continuous positive airway pressure, CPAP, or bilevel airway pressure) for treatment of sleep apnea or sleep disordered breathing is allowed if settings have been stable for at least 1 month prior to study entry, and is approved by the medical monitor.
•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 previous hematopoietic stem cell transplant (HSCT)
•Has received previous treatment with BMN 110
+7 more criteria

Locations (11)

Oakland, California, United States

Chicago, Illinois, United States

New York, New York, United States

Houston, Texas, United States

Calgary, Alberta, Canada

Toronto, Ontario, Canada

Montreal, Quebec, Canada

Sherbrook, Quebec, Canada

Hamburg, Germany

Belfast, Northern Ireland, United Kingdom

Key Dates

Start Date

April 1, 2012

Primary Completion Date

November 1, 2014

Completion Date

November 1, 2014

Last Updated

February 1, 2016

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA

Interventions

BMN 110

DRUG

BMN 110

DRUG

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

NCT03632213

Mucopolysaccharidosis IV AMucopolysaccharidosis VI+7 more

View study

COMPLETEDPHASE3

BMN 110 Phase 3B in Australian Patients

NCT01966029

Mucopolysaccharidosis IVA (Morquio A Syndrome)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 1, 2016Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Inclusion Criteria

Exclusion Criteria

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

BMN 110 Phase 3B in Australian Patients

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)