A Study to Evaluate the Safety and Efficacy of BMN 110 in MPS IVA Patients Less Than 5 Years of Age

Phase 1

• Conditions: Mucopolysaccharidosis Type IVA; MedDRA version: 14.1Level: PTClassification code 10028095Term: Mucopolysaccharidosis IVSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2011-003197-84-IT
• Lead Sponsor: BIOMARIN PHARMACEUTICAL INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-03-12
• Last Update Posted: 31 January 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1- Less than 5 years of age. 2-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. 3-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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 study 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 patient at high risk of poor treatment compliance or of not completing the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified