Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT ID: NCT01515956

Last Updated: 2017-08-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-10-31
• Study Completion Date: 2016-02-29

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.

Conditions

Mucopolysaccharidosis IVA; Morquio A Syndrome; MPS IVA

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

BMN 110 Weekly

Group Type: EXPERIMENTAL

BMN 110

Intervention Type: DRUG

Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 208 weeks.

Interventions

BMN 110

Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 208 weeks.

Intervention Type: DRUG

Other Intervention Names

N-acetylgalactosamine-6-sulfatase; N-acetylgalactosamine-6-sulfate; sulfatase; galactose-6-sulfatase; GALNS; enzyme replacement therapy; ERT

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.

• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Debra Lounsbury

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

Oakland, California, United States

Manhasset, New York, United States

Monza, , Italy

Taipei, , Taiwan

Central Manchester, , United Kingdom

Countries

United States; Italy; Taiwan; United Kingdom

References

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.

Reference Type: DERIVED

PMID: 26331768 (View on PubMed)

Other Identifiers

MOR-007

Identifier Type: -

Identifier Source: org_study_id