Mucopolysaccharidosis IVA, also known as Morquio A Syndrome
MPS IVA (Morquio A syndrome) results from a lower amount of an enzyme called N-acetylgalactosamine-6 sulfatase (GALNS) than usual. Because of this, people with MPS IVA cannot break down a material called keratan sulfate (KS) in many tissues and organs. The buildup of KS causes many problems such as difficulty walking, trouble breathing, short height, abnormal chest shape, and bone and joint problems. Other symptoms may include hearing loss, corneal clouding and heart valve disease. Morquio A syndrome is estimated to occur in 1 in 200,000 to 300,000 babies. It is inherited in an autosomal recessive manner, affects males and females equally, and in most cases, both parents of an affected child are asymptomatic carriers of the disease.
Program Overview
BioMarin’s experimental drug for Morquio A syndrome is an enzyme replacement of GALNS (called BMN 110), which is designed to clear keratan sulfate from the lysosome. BMN 110 is being studied to determine if it is safe, if it will slow the progression of the disease and if it will improve some of the symptoms.
BMN 110 at a Glance
- BioMarin started BMN 110 clinical studies in humans in 2009 to evaluate safety and efficacy
- Assigned the Orphan Drug designation in the United States and European Union
Patient Educational Information
Understanding Clinical Studies that Involve Enzyme Replacement Therapy (ERT ) (video):
In this video, you will hear from a leading health care provider, who is an expert in Morquio A Syndrome about the key elements of participating in an enzyme replacement therapy clinical study. The expert also answers important questions that you may have about ERT clinical studies. The video features Dr. Chris Hendriksz and his study team at Birmingham Children’s Hospital in the United Kingdom (UK). They are joined by patients and their families who share their stories about participating in ERT clinical studies.
Current Clinical Trials
1. A Phase 2, Open Label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation (MOR-006)
This study will evaluate the safety and efficacy of BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome) who have limited ability to walk.
Key Study Facts:
- Enrolling MPS IVA patients greater than 5 years of age who meet protocol criteria for the 6 minute walk test by invitation only.
- Participants will receive 2 mg/kg weekly dosing of study drug via infusion for 48 weeks.
- Most visits will consist of one full day, and some visits will require several days in a row.
For additional information about the study, including details about participating centers, please see click here.
2. A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio A Syndrome) (MOR-001)
This study called MorCAP is designed to provide a better understanding of MPS IVA by measuring function of various aspects of the disorder including walking, climbing stairs and breathing in affected patients.
Key Study Facts:
- Enrolling MPS IVA patients of all ages
- The study is observational (ie there is no study drug involved)
- The study will include 1 or more scheduled doctor visits
For additional information about the study, including details about participating centers, please click here
3. A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) MOR-004
This study is evaluating whether the study drug BMN 110 is safe and effective in treating the symptoms of MPS IVA and whether it has side effects. In particular, the study will evaluate the patients’ endurance in walking and climbing stairs.
Key Study Facts:
- Enrolling MPS IVA patients at least 5 years of age. Enrollment complete.
- Must meet the study entrance requirements for the 6-minute walk test
- Participants will receive weekly dosing of study drug or placebo via infusion for 24 weeks, and the participants may have the option to take part in an extension study
- The study will include 27 scheduled doctor visits. Most visits will consist of one full day, and some visits will require that you come for several days in a row
For additional information about the study, including details about participating centers, please click here
4. A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) MOR-005
This Phase 3 extension study will evaluate the long-term safety and efficacy of BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
Key Study Facts:
- Enrolling MPS IVA patients at least 5 years of age who have completed the phase 3 study (MOR-004)
- Participants will receive 2 mg/kg weekly or every other weekly dosing of study drug via infusion until the MOR-004 study is unblinded and the optimal dose is selected. All subjects will then be treated with the optimal dose for up to approximately 5 years or until the drug is approved,
- Most visits will consist of one full day, and some visits will require that you come for several days in a row.
For additional information about the study, including details about participating centers, please see click here
5. 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) (MOR-007)
This Phase 2 study will evaluate the safety and efficacy of BMN 110 in pediatric patients less than 5 years of age with mucopolysaccharidosis IVA (Morquio A Syndrome).
Key Study Facts:
- Enrolling MPS IVA patients under 5 years of age at the time of BMN 110 dosing by invitation only.
- Participants will receive 2 mg/kg weekly dosing of study drug via infusion for up to 52 weeks.
- Most visits will consist of one full day, and some visits will require several days in a row.
For additional information about the study, including details about participating centers, please see click here
6. A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (MOR-100)
This study will evaluate the safety and efficacy of BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
Key Study Facts:
- Enrolling MPS IVA patients who have participated in a previous BMRN sponsored clinical study of BMN 110 (except for MOR-004). Participants will receive 2 mg/kg weekly dosing of study drug via infusion for up to approximately 240 weeks,
- Most visits will consist of one full day, and some visits will require several days in a row.
For additional information about the study, including details about participating centers, please see click here
7. A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) (MOR-008)
This study will evaluate the safety and efficacy of BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
Key Study Facts:
- Enrolling MPS IVA patients greater than 7 years of age who meet protocol criteria for the 6 minute walk test, who do not require the use of supplemental oxygen, and who have not used a ventilator for the 3 months prior to study entry.
- Participants will receive 2 or 4 mg/kg weekly dosing of study drug via infusion for 27 weeks.
- Most visits will consist of one full day, and some visits will require several days in a row.
For additional information about the study, including details about participating centers, please see click here
Completed Clinical Studies
A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio A Syndrome), please click here