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

Morquio A Syndrome Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01515956
• Other study ID #: MOR-007
• Status: Completed
• Phase: Phase 2
• First received: December 22, 2011
• Last updated: December 19, 2016
• Start date: October 2011
• Est. completion date: June 2016

Study information

• Verified date: December 2016
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review BoardUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyUnited Kingdom: National Institute for Health ResearchUnited Kingdom: Research Ethics CommitteeItaly: The Italian Medicines AgencyItaly: Ethics CommitteeTaiwan : Food and Drug AdministrationTaiwan: Department of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: June 2016
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 5 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Morquio A Syndrome
• MPS IVA
• Mucopolysaccharidoses
• Mucopolysaccharidosis IV
• Mucopolysaccharidosis IVA
• Syndrome

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Italy,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Descriptive summary of clinical safety assessments	Safety will be determined by the following factors: Number and severity of adverse events in participants. Clinically significant changes in any of the following assessments from Baseline: Vital Signs, echocardiogram, ECG, immunogenicity tests, physical and neurological examinations, standard clinical laboratory tests, concomitant medications, and cervical spine radiography.	Up to 208 weeks/ETV	Yes
Secondary	Change in Urinary Keratan Sulfate measures over time		Baseline, and weeks: 2, 4, 8, 13, 26, 39, 52, 78, 104, 130, 156, 182, 208/ETV	No
Secondary	Change in patient growth over time	Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities.	Baseline and Weeks: 13, 26, 39, 52,78, 104, 130, 156, 182, 208/ETV	No