Clinical trial NCT01515956: Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Official title: A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Study type: INTERVENTIONAL
Overall status: COMPLETED
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
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2012-01-24
Enrollment: 15
Sex: ALL
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks.


Eligibility criteria: Inclusion Criteria:

* Less than 5 years of age at the time of the first study drug infusion
* 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
* Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.

Exclusion Criteria:

* Previous hematopoietic stem cell transplant (HSCT).
* Previous treatment with BMN 110.
* Known hypersensitivity to any of the components of BMN 110.
* Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period.
* Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
* 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 safety as determined by the Investigator.
* Any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study.

Primary outcomes:
measure: To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion
timeFrame: 52 weeks
Secondary outcomes:
measure: Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures
timeFrame: Baseline to Week 52
measure: Change From Baseline in Normalized Growth Rate Z-Scores
timeFrame: Baseline to Week 52
Other outcomes:
Locations:
city: Oakland
country: United States
state: California
city: Manhasset
country: United States
state: New York
city: Monza
country: Italy
city: Taipei
country: Taiwan
city: Central Manchester
country: United Kingdom
Study officials:
affiliation: BioMarin Pharmaceutical
name: Debra Lounsbury
role: STUDY_DIRECTOR
References:
citation: Jones SA, Bialer M, Parini R, Martin K, Wang H, Yang K, Shaywitz AJ, Harmatz P. Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y. Pediatr Res. 2015 Dec;78(6):717-22. doi: 10.1038/pr.2015.169. Epub 2015 Sep 2.