Clinical trials: MPS VI
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
- Trial name
- NCT00299000
- Testing
- Naglazyme
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 4 people
- Sponsor
- BioMarin Pharmaceutical
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Mucopolysaccharidosis (MPS)
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of…
- Trial name
- NCT02095015
- Where
- 14 sites in United States, Canada and 5 more
- Volunteers
- 159 people
- Sponsor
- Shire
Mucopolysaccharidosis VI
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with…
- Trial name
- NCT00048711
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals…
- Trial name
- NCT00214773
- Where
- 50 sites in United States, Australia and 11 more
- Volunteers
- 237 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis VI
Study of rhASB in Patients With Mucopolysaccharidosis VI
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
- Trial name
- NCT00104234
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis VI
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in…
- Trial name
- NCT00048620
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Sponsor
- BioMarin Pharmaceutical
MPS VI
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients.
- Trial name
- NCT01387854
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 59 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis VI
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk…
- Trial name
- NCT00067470
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis VI
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs).
- Trial name
- iMProveS (NCT03370653)
- Testing
- Odiparcil
- Where
- 4 sites in France, Germany and 2 more
- Volunteers
- 20 people
- Sponsor
- Inventiva Pharma
Lysosomal Storage Disease, Lung Diseases
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Maroteaux-Lamy disease from blood
- Conditions
- Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia
- Trial name
- BioMaroteaux (NCT01458613)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock