Clinical trials: MPS IV
Morquio Syndrome A, Maroteaux Lamy Syndrome
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
The purpose of this study is to identify patients with Morquio syndrome type A (MPS IVA) and Maroteaux-Lamy syndrome (MPS VI) who may have been missed or misdiagnosed due to atypical clinical features, a milder course, and/or…
- Conditions
- Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
- Trial name
- NCT01961518
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Greenwood Genetic Center
MPS IVA, MPS VI
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI.
- Trial name
- NCT05845749
- Testing
- Vosoritide Injection [Voxzogo]
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all…
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Trial name
- NIFAMA (NCT05284006)
- Testing
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Nemours Children's Clinic
Mucopolysaccharidosis IV A, Morquio A Syndrome
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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).
- Conditions
- Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01415427
- Testing
- BMN 110 - Weekly, BMN 110 - Every Other Week
- Where
- 43 sites in United States, Argentina and 18 more
- Volunteers
- 173 people
- Sponsor
- BioMarin Pharmaceutical
Hurler Syndrome
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an…
- Trial name
- NCT01173016
- Testing
- Laronidase
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Mucopolysaccharidosis Type IVA
Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
The purpose of this study is to find out more about some of the unusual chemicals (called oligosaccharides) that can occur naturally as a result of processes in the body.
- Trial name
- NCT01733615
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Mucopolysaccharidosis IV A
Natural History of Atypical Morquio A Disease
Mucopolysaccharidosis IVA (MPS IVA) (or Morquio A disease) is a rare recessive autosomal lysosomal storage disorder caused by deficiency of N-acetylgalactosamine-6-sulfatase (GALNS) resulting in accumulation of the…
- Trial name
- NCT03204370
- Testing
- Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
- Where
- France
- Volunteers
- 7 people
- Sponsor
- GOIZET
Mucopolysaccharidosis IV, Morquio A Disease
Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
Mucopolysaccharidosis Type IVa (MPS IVa, Morquio Disease) is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme galactose-6-sulfatase.
- Trial name
- NCT02153255
- Where
- United Kingdom
- Sponsor
- Birmingham Women's and Children's NHS Foundation Trust
Morquio A Syndrome, Mucopolysaccharidosis IV A
Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Mucopolysaccharidosis IV, also known as MPS IV or Morquio disease, is a rare autosomal recessive genetic lysosomal storage disorder.
- Trial name
- MAPLE (NCT02208661)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Nadia Ali, PhD
Kidney Transplantation
Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Renal Transplant Patients
The aim of MYFORMS is to assess efficacy and safety on clinical outcomes of EC-MPS in combination with Cyclosporine microemulsion (CsA-ME) in kidney transplant recipients.
- Trial name
- NCT00239083
- Testing
- Enteric-Coated Mycophenolate Sodium (EC-MPS)
- Where
- Switzerland
- Volunteers
- 40 people
- Sponsor
- Novartis
MPS IVA, Morquio Syndrome
Gait Analysis in MPS IVA
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
- Trial name
- NCT01920828
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Oregon Health and Science University
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
- Trial name
- LSD Registry (NCT05619900)
- Testing
- There is no intervention
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- University of California, San Francisco
Morquio Syndrome, Accumulation of Mucopolysaccharides
Biomarker for Morquio Disease (BioMorquio)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Morquio disease from plasma
- Conditions
- Morquio Syndrome, Accumulation of Mucopolysaccharides, Morquio Syndrome A, Morquio B Disease
- Trial name
- BioMorquio (NCT01457456)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
BACKGROUND/OBJECTIVE: Quantitative urine screening for mucopolysaccharides (MPS) has been the primary method for detecting mucopolysaccharidoses in children.
- Trial name
- NCT01707433
- Testing
- Enzyme testing
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Children's Hospitals and Clinics of Minnesota
Mucopolysaccharidosis I, Mucopolysaccharidosis II
MPS (RaDiCo Cohort) (RaDiCo-MPS)
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mucopolysaccharidosis IV
- Trial name
- NCT06036693
- Where
- 23 sites in France
- Volunteers
- 1,000 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
MPS IV A, Mucopolysaccharidosis IVA
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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).
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Trial name
- NCT01242111
- Testing
- BMN 110
- Where
- 4 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Mucopolysaccharidoses (MPS) are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of…
- Conditions
- Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidoses, MPS IV A
- Trial name
- NCT03632213
- Testing
- Losartan
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
MPS I, MPS II
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses…
- Conditions
- MPS I, MPS II, MPS IVA, MPS VI
- Trial name
- PEARL (NCT04532047)
- Testing
- Aldurazyme (laronidase)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, San Francisco
Mucopolysaccharidosis IVA, Morquio A Syndrome
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
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.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01697319
- Testing
- BMN 110
- Where
- 7 sites in United States, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical