Search clinical trials for: Mucopolysaccharidosis VI

Total 463575 results

University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

MRS to Determine Neuroinflammation and Oxidative Stress in MPS I

Mucopolysaccharidosis Type I

United States
Inventiva Pharma

Completed

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil (iMProveS)

Mucopolysaccharidosis VI

United Kingdom, France, Germany, Portugal
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Neurobehavioral Phenotypes in MPS III

Hurler Syndrome | Sanfilippo Syndrome Type A | Sanfilippo Syndrome Type B

United States
Ultragenyx Pharmaceutical Inc

Completed

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

Spain, United States, Portugal
Oregon Health and Science University
University of Utah; BioMarin Pharmaceutical

Completed

Gait Analysis in MPS IVA

MPS IVA | Morquio Syndrome

United States
University of Chicago
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Krabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)

United States
BioMarin Pharmaceutical

Completed

BMN 110 Phase 3B in Australian Patients (MOR-AUS)

Mucopolysaccharidosis IVA (Morquio A Syndrome)

Australia
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Mucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VII

United States, Canada
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant w/Laronidase for Hurler

Mucopolysaccharidosis I | Hurler Syndrome

United States
Masonic Cancer Center, University of Minnesota

Completed

HSCT for High Risk Inherited Inborn Errors

Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell Disease

United States
Ultragenyx Pharmaceutical Inc

Completed

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Mucopolysaccharidosis Type 7

United Kingdom
University of Texas Southwestern Medical Center

Withdrawn

Effects of Growth Hormone in Chronically Ill Children

HIV Infections | Cerebral Palsy | Crohn Disease | Hurler Syndrome | Juvenile Rheumatoid Arthritis

United States
Masonic Cancer Center, University of Minnesota

Completed

Intrathecal Enzyme Replacement for Hurler Syndrome

Hurler Syndrome

United States
Masonic Cancer Center, University of Minnesota

Terminated

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Hurler Syndrome | Mucopolysaccharidosis Type IH | MPS I

United States
ArmaGen, Inc

Completed

Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

Mucopolysaccharidosis I
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Mucopolysaccharidosis II

United States, Germany, Philippines
Alexion Pharmaceuticals

Terminated

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo B Syndrome)

Spain, United States, Brazil, United Kingdom, Portugal, Italy
Lundquist Institute for Biomedical Innovation at...

Terminated

Clinical Trial of Growth Hormone in MPS I, II, and VI

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI

United States
Alexion Pharmaceuticals

Completed

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

Mucopolysaccharidosis IIIB

United States, United Kingdom, Spain
Alexion Pharmaceuticals

Terminated

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously (CL01-T)

Mucopolysaccharidosis III, Type B (MPS IIIB) | Sanfilippo B

United Kingdom
Nadia Ali, PhD
BioMarin Pharmaceutical

Completed

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) (MAPLE)

Morquio A Syndrome | Mucopolysaccharidosis IV A

United States
Masonic Cancer Center, University of Minnesota

Completed

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Mucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy Syndrome

United States
Children's Hospital of Eastern Ontario

Completed

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

MPS I | MPS IV

Canada
Mackay Memorial Hospital
BioMarin Pharmaceutical

Unknown

A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis

Mucopolysaccharidoses
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplantation for Hurler

Mucopolysaccharidosis I | Mucopolysaccharidosis VI | Mannosidosis | Mucolipidosis Type II (I-cell Disease)

United States
Masonic Cancer Center, University of Minnesota

Terminated

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B

United States
University Hospital Heidelberg

Completed

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Mucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Coping Behavior | Mucopolysaccharidosis Type III | Behavior Disorders

Germany
Manchester Royal Eye Hospital
BioMarin Pharmaceutical

Unknown

New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis

Mucopolysaccharidoses

United Kingdom
GlaxoSmithKline

Completed

A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

Mucopolysaccharidosis II

Japan
Lundquist Institute for Biomedical Innovation at...

Completed

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

Mucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI

United States
BioMarin Pharmaceutical

Completed

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

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

Taiwan, United States, Italy, United Kingdom
LYSOGENE

Completed

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Mucopolysaccharidosis Type III A | Sanfilippo Disease Type A

France
Joyce Fox
Ultragenyx Pharmaceutical Inc

No longer available

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Mucopolysaccharidosis Type 7

United States
Alexion Pharmaceuticals

Completed

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo Syndrome)

Spain, Netherlands, United Kingdom, United States, Brazil
Swiss Federal Institute of Technology
University for Development Studies, Tamale, Ghana

Completed

Comparison of Iron Absorption From Extruded FePP-fortified Rice Containing Zinc, Citric Acid/Trisodium Citrate and EDTA (MM_Rice)

Iron Deficiency and/or Anemia

Switzerland, Ghana
Birmingham Women's and Children's NHS Foundation...
BioMarin Pharmaceutical

Withdrawn

Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa

Mucopolysaccharidosis IV | Morquio A Disease

United Kingdom
Alexion Pharmaceuticals

Completed

Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging

MPS IIIB (Sanfilippo B Syndrome)

United Kingdom
Children's Hospital Los Angeles

Completed

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Granuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler Disease

United States
BioMarin Pharmaceutical

Terminated

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United States, United Kingdom, Canada, Germany
Green Cross Corporation

Unknown

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Mucopolysaccharidosis II
Patricia I. Dickson, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaborators

Completed

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Mucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Cognitive Decline | Hurler-Scheie Syndrome

United States
BioMarin Pharmaceutical

Terminated

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

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United Kingdom, United States, Germany
Nationwide Children's Hospital
Sanfilippo Children's Research Foundation; The Sanfilippo Research Foundation; The Children's Medical Research Foundation

Completed

Natural History Studies of Mucopolysaccharidosis III

Mucopolysaccharidosis Type IIIA | Mucopolysaccharidosis Type IIIB

United States
BioMarin Pharmaceutical

Terminated

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

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

United Kingdom
Greenwood Genetic Center
Shriners Hospitals for Children; BioMarin Pharmaceutical

Completed

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

MPS IVA | Maroteaux Lamy Syndrome | MPS VI | Morquio Syndrome A

United States
Children's Hospitals and Clinics of Minnesota
BioMarin Pharmaceutical; Gillette Children's Specialty Healthcare; Greenwood...

Completed

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

Mucopolysaccharidosis VI | Mucopolysaccharidosis IV A

United States
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

Mucopolysaccharidosis I | Hurler-Scheie Syndrome | Hurlers Syndrome

United States, Canada, Germany
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

Mucopolysaccharidosis I | Scheie Syndrome | Hurler Syndrome | Hurler-Scheie Syndrome

Netherlands, France, Germany, United Kingdom
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Mucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie Syndrome

United States, Canada, United Kingdom, Brazil, Germany, Italy, Netherlands
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Mucopolysaccharidosis I | Scheie Syndrome | Hurler's Syndrome | Hurler-Scheie Syndrome

Brazil, Canada

Search clinical trials for: Mucopolysaccharidosis VI

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