Clinical Trials on Mucopolysaccharidosis Disorders

Total 145105 results

Green Cross Corporation

Completed

A Study of GC1111 in Hunter Syndrom Patients

Hunter Syndrome

Korea, Republic of
Green Cross Corporation

Unknown

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Hunter Syndrome

Korea, Republic of
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
Mackay Memorial Hospital
Sanofi Taiwan Co. Ltd

Recruiting

The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan

MPS I

Taiwan
Children's Hospital of Eastern Ontario

Completed

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

MPS I | MPS IV

Canada
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
CENTOGENE GmbH Rostock

Terminated

Biomarkers for Hunter Syndrome (BioHunter)

Mucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal Syndrome

Mexico
Shire
Covance; PRA Health Sciences; PharmaNet

Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

Brazil, Poland, Taiwan
University Hospital, Tours

Completed

Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis (RespiGAG)

Mucopolysaccharidoses

France
University of California, San Francisco

Recruiting

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

MPS IVA | MPS VI

United States
BioMarin Pharmaceutical

Completed

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

MPS VI

United States, Australia, France, Brazil, Germany, Portugal, United Kingdom
Albert Einstein College of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)

Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions

United States
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
Lysosomal and Rare Disorders Research and Treatment...
Team Sanfilippo

Recruiting

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

Sanfilippo Syndrome | MPS3

United States
Allievex Corporation

Enrolling by invitation

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

MPS III B

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

Completed

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Mucopolysaccharidoses

United States
Lundquist Institute for Biomedical Innovation at...
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI

Mucopolysaccharidoses

United States
Sanofi

Completed

A First-in-human Single and Repeated Dose Escalation Study of SAR442501 in Healthy Adults Subjects

Osteochondrodysplasia

United States
BioMarin Pharmaceutical

Completed

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

MPS IV A

United Kingdom
Patricia I. Dickson, M.D.
The Ryan Foundation

Terminated

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Mucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Spinal Cord Compression | Hurler-Scheie Syndrome

Finland, United States
Masonic Cancer Center, University of Minnesota
BioMarin Pharmaceutical

Terminated

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Maroteaux-Lamy Syndrome

United States
BioMarin Pharmaceutical

Completed

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

MPS IV A

United States, Canada, France, United Kingdom, Taiwan, Argentina, Colombia, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, Germany, Portugal, Italy, Qatar
CENTOGENE GmbH Rostock

Completed

Establishment of Human Cellular Disease Models for Morquio Disease (IPSMORQUIO)

Morquio Disease

Pakistan
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
Masonic Cancer Center, University of Minnesota

Completed

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Hurler Syndrome

United States
Samsung Medical Center

Not yet recruiting

Natural History Observational Study of MPS IIIa in SMC

Sanfilippo Syndrome A
Shire

Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Sanfilippo Syndrome

United States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
UniQure Biopharma B.V.
Institut Pasteur; Venn Life Sciences

Completed

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Sanfilippo Syndrome B

France
Sanofi

Recruiting

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia (upreACH-2)

Osteochondrodysplasia

Australia, Spain, Korea, Republic of, China, Italy
Phoenix Nest
National Institute of Neurological Disorders and Stroke (NINDS)

Enrolling by invitation

A Natural History Study of Sanfilippo Syndrome Type D (MPSIIID)

Sanfilippo Syndrome Type D

United States
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Turkey
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Germany, Turkey
Shire

Terminated

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome

Netherlands, United Kingdom
Oregon Health and Science University
University of Utah; BioMarin Pharmaceutical

Completed

Gait Analysis in MPS IVA

MPS IVA | Morquio Syndrome

United States
Shire

Completed

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome Type A

United States
Aldagen

Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

Mucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage Diseases

United States
Shire

Completed

Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Hunter Syndrome

Mexico
Takeda
Takeda Development Center Americas, Inc.

Active, not recruiting

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Hunter Syndrome

United States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
Children's Hospital of Orange County
Ultragenyx Pharmaceutical Inc

Recruiting

Cardiovascular Structure and Function in the Mucopolysaccharidoses

Inflammation | Mucopolysaccharidoses | Cardiac Disease | Carotid Disease

United States
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Sly Disease (MPS VII) (BioSly) (BioSly)

Developmental Delay | Splenomegaly | Hepatomegaly | Skeletal Abnormalities

Germany, India, Sri Lanka, Egypt
Clínica de Hidroterapia e Recuperacao Funcional
Integrated Faculty of Recife

Terminated

Morquio's Syndrome: a Case Study

Morquio's Disease
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Green Cross Corporation

Completed

Safety and Efficacy of Hunterase (GC1111)

Hunter Syndrome

Korea, Republic of
St. Jude Children's Research Hospital

Recruiting

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Mucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, Hematologic

United States
Shire

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Hunter Syndrome

United States, United Kingdom
Takeda

Recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Hunter Syndrome

Canada, France
Shire

Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
Shire

Completed

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire

Completed

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom

Clinical Trials on Mucopolysaccharidosis Disorders

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Burundi

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations