Clinical trials: MPS IVA
MPS IV A, Mucopolysaccharidosis IVA
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
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…
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Trial name
- NCT00787995
- Where
- 15 sites in United States, Argentina and 8 more
- Volunteers
- 353 people
- Sponsor
- BioMarin Pharmaceutical
MPS I, MPS IV A
Cardiac Structure and Function in MPS
The purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS).
- Conditions
- MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
- Trial name
- NCT07361536
- Where
- 3 sites in United States
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Orange County
MPS IV A
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed…
- Trial name
- NCT00884949
- Testing
- BMN 110
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IVA, Morquio A Syndrome
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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…
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01515956
- Testing
- BMN 110
- Where
- 5 sites in United States, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IVA, Morquio A Syndrome
BMN 110 US Expanded Access Program
The Expanded Access Program (EAP) is an open-label, multicenter program to: 1. Provide patients who have been diagnosed with Mucopolysaccharidosis IVA (MPS IVA) access to BMN 110 until commercial product is available 2.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01858103
- Testing
- BMN 110
- Where
- 25 sites in United States, Puerto Rico
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the…
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Trial name
- MARS (NCT02294877)
- Testing
- Vimizim® (elosulfase alfa)
- Where
- 79 sites in United States, Australia and 15 more
- Volunteers
- 418 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IVA, Morquio A Syndrome
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
The primary objective of this study was 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 196 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01609062
- Testing
- BMN 110
- Where
- 11 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Neurobehavioral function and quality of life are compromised in many patients with mucopolysaccharidosis (MPS) disorders. The long-term goals of this research are to: 1) more accurately inform patients/parents regarding potential…
- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV
- Trial name
- NCT01870375
- Where
- 4 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- University of Minnesota