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MPS IVA Clinical Trials

MPS IVA, also known as Morquio A, is a rare inherited disease that affects major organ systems in the body. The condition is a form of mucopolysaccharidosis, which is a type of lysosomal storage disorder.

Learn more about our MPS IVA clinical trials below.

Elosulfase alfa

Studies

Study 110-504 Active, Not Recruiting

A Multicenter, Multinational, Observational Morquio A Registry Study in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome)

Investigational therapeutic

None

Status

Active, Not Recruiting

Study Type

Observational, Non-Interventional Registry

Study MOR-005 Complete

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

Evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome)

Investigational therapeutic

Elosulfase alfa (BMN 110)

Status

This study has been completed

Study Type

Phase 3

Study BMN-110 Complete

Open-label Phase 2 study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

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

Investigational therapeutic

Elosulfase alfa (BMN 110)

Status

This study has been completed

Study Type

Phase 2

Study MOR-007 Complete

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

Evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks

Investigational therapeutic

Elosulfase alfa (BMN 110)

Status

This study has been terminated

Study Type

Phase 2

Study MOR-001 Complete

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time

Investigational therapeutic

Elosulfase alfa (BMN 110)

Status

This study has been terminated

Study Type

Observational

Study MOR-100 Complete

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA)

Investigational therapeutic

Elosulfase alfa (BMN 110)

Status

This study has been terminated

Study Type

Phase 2

Study MOR-008 Complete

Safety and Exercise Study of BMN 110 for Morquio A Syndrome

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Indication

MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

Goal

Evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 192 weeks. Secondary objectives will investigate the effect of the two doses on exercise capacity for up to 192 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 will be assessed.

Investigational therapeutic

Elosulfase alfa (BMN 110)

Status

This study has been terminated

Study Type

Phase 2