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

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)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-003197-84-GB

Enrollment

Registered

2011-09-13

Start date

2011-11-22

Completion date

Unknown

Last updated

2019-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis Type IVA MedDRA version: 14.1 Level: PT Classification code 10028095 Term: Mucopolysaccharidosis IV System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: BMN 110 Product Code: N/A Pharmaceutical Form: Solution for infusion INN or Proposed INN: N/A CAS Number: 9025-60-9

Eligibility

Sex/Gender

All

Inclusion 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. 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

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.

Design outcomes

Primary

Measure	Time frame
Timepoint(s) of evaluation of this end point: Assessed at Screening and each visit; Main Objective: 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 patients less than 5 years of age. For the extension Phase: To evaluate the long-term safety of BMN 110 at a dose of 2.0 mg/kg/week in patients with MPS IVA less than 5 years of age at enrollment. ; Secondary Objective: • To evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age. • To evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age. For the extension phase of the study: • To evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary KS levels in MPS IVA patients less than 5 years of age at enrollment. • To evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age at enrollment. ;Primary end point(s): Evaluate safety and tolerability of infusions of BMN 110 at a dose of 2.0 mg/kg/week over a maximum of 209 week period (52 weeks in the initial clinical phase, and up to 156 doses plus 1 week of assessments in the extension phase) in MPS IVA patients less than 5 years of age (at the time of administration of the first dose of study drug).	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: Assessed at Screening and each visit; Main Objective: 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 patients less than 5 years of age. For the extension Phase: To evaluate the long-term safety of BMN 110 at a dose of 2.0 mg/kg/week in patients with MPS IVA less than 5 years of age at enrollment. ; Secondary Objective: • To evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age. • To evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age. For the extension phase of the study: • To evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary KS levels in MPS IVA patients less than 5 years of age at enrollment. • To evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age at enrollment. ;Primary end point(s): Evaluate safety and tolerability of infusions of BMN 110 at a dose of 2.0 mg/kg/week over a maximum of 209 week period (52 weeks in the initial clinical phase, and up to 156 doses plus 1 week of assessments in the extension phase) in MPS IVA patients less than 5 years of age (at the time of administration of the first dose of study drug).

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Secondary

Measure	Time frame
Secondary end point(s): * Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age (at the time of administration of the first dose of study drug) * Evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age (at the time of administration of the first dose of study drug) ; Timepoint(s) of evaluation of this end point: • Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age: Assessed at Baseline, Weeks 2, 4, 8, 13, 26, 39, and 52 and then every 26 weeks up to week 208. • Evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age: Assessed at Baseline and every 13 weeks up to week 52 and then every 26 weeks up to week 208.	—

Measure

Time frame

Secondary end point(s): * Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age (at the time of administration of the first dose of study drug) * Evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age (at the time of administration of the first dose of study drug) ; Timepoint(s) of evaluation of this end point: • Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age: Assessed at Baseline, Weeks 2, 4, 8, 13, 26, 39, and 52 and then every 26 weeks up to week 208. • Evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age: Assessed at Baseline and every 13 weeks up to week 52 and then every 26 weeks up to week 208.

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Countries

Italy, United Kingdom, United States

Contacts

Public ContactClinical Trials Information

BioMarin Pharmaceutical Inc.

clinicaltrials@bmrn.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026