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Vimizim® for Morquio A Syndrome (Mucopolysaccharidosis IVA, or MPS IVA)

Current Clinical Trials: MPS IVA

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

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

Investigational Therapeutic: BMN 110

Study Type: Phase 3

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).

Status: This study is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

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

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

Investigational Therapeutic: BMN 110

Study Type: Phase 2

Goal: 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.

Status: This study is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Completed Clinical Trials: MPS IVA

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

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

Investigational Therapeutic: BMN 110

Study Type: Phase 2

Goal: The primary objective of this study is to 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.
Status: This study has been terminated

Additional information: View on Clinicaltrials.gov

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

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

Investigational Therapeutic: BMN 110

Study Type: Observational

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.

Status: This study has been terminated

Additional information: View on Clinicaltrials.gov

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

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

Investigational Therapeutic: BMN 110

Study Type: Phase 2

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)

Status: This study has been terminated

Additional information: View on Clinicaltrials.gov

Study: Safety and Exercise Study of BMN 110 for Morquio A Syndrome

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

Investigational Therapeutic: BMN 110

Study Type: Phase 2

Goal: The primary objective of this study is to 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

Status: This study has been terminated

Additional information: View on Clinicaltrials.gov

Clinical Trial

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

Clinical Trial

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

Clinical Trial

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

Clinical Trial

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

Clinical Trial

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

Clinical Trial

Safety and Exercise Study of BMN 110 for Morquio A Syndrome
Read more »

BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI) PKU, LEMS

Products

Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

Global Medical Information

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