Search clinical trials for: Mucopolysaccharidosis II

Total 31486 results

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
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
Lundquist Institute for Biomedical Innovation at...

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI

United States
CENTOGENE GmbH Rostock

Terminated

Biomarkers for Hunter Syndrome (BioHunter)

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

Mexico
Allievex Corporation

Active, not recruiting

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type IIIB | MPS III B

Germany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
Hospital de Clinicas de Porto Alegre
The Isaac Foundation

Active, not recruiting

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Mucopolysaccharidoses | Mucopolysaccharidosis VI | Morquio A Syndrome | Mucopolysaccharidosis IV A | MPS IV A | MPS VI | MPS - Mucopolysaccharidosis | Morquio Syndrome A | Morquio Syndrome

Brazil
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Mucopolysaccharidosis II

Japan
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Japan
Allievex Corporation

Active, not recruiting

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB

United States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Mucopolysaccharidosis II | MPS II

United States
REGENXBIO Inc.

Withdrawn

Mucopolysaccharidosis Type II Observational

Mucopolysaccharidosis II

United States, Canada
Abeona Therapeutics, Inc

Terminated

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

Mucopolysaccharidosis III-B

United States, Germany, France
Takeda

Completed

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Mucopolysaccharidosis (MPS)

Brazil
Abeona Therapeutics, Inc

Terminated

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB (MPSIIIB)

Mucopolysaccharidosis Type 3 B

United States, Germany, France, Spain
REGENXBIO Inc.

Completed

Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis II

Brazil, United States, United Kingdom
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
Fondazione Telethon

Active, not recruiting

Gene Therapy in Patients With Mucopolysaccharidosis Disease

Mucopolysaccharidosis Type VI

Italy, 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
LYSOGENE

Completed

Natural History Study of Patients With MPS IIIA

Mucopolysaccharidosis IIIA

Germany, Brazil, United Kingdom, France, Netherlands
LYSOGENE

Active, not recruiting

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Mucopolysaccharidosis Type IIIA

Germany, United States, United Kingdom, France, Netherlands
REGENXBIO Inc.

Enrolling by invitation

Long-term Follow-Up for RGX-121

Mucopolysaccharidosis II

United States
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
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
Shire

Completed

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Mucopolysaccharidosis (MPS)

United Kingdom, Netherlands
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 Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome Type A

United States
Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

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

Terminated

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Sanfilippo Syndrome | Mucopolysaccharidosis (MPS)

United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
Shire

Completed

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Hunter Syndrome | Mucopolysaccharidosis II (MPS II)

United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
Shire

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
Shire

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Hunter Syndrome

United States, United Kingdom
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
Shire

Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome

United Kingdom, United States, Brazil
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
BioMarin Pharmaceutical

Completed

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
Shire

Completed

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Hunter Syndrome | Mucopolysaccharidosis (MPS)

United States, Argentina, Mexico, Spain, United Kingdom
Shire

Terminated

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)

United States, Canada, United Kingdom, Turkey, Italy, Mexico, Spain
Shire

Completed

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Sanfilippo Syndrome Type B

United States, United Kingdom, Germany
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
AO GENERIUM

Completed

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Metabolic Diseases | Mucopolysaccharidosis Type II

Russian Federation
Allievex Corporation

Completed

A Treatment Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | MPS III B

United States, Taiwan, Spain, Colombia, Germany, United Kingdom, Turkey
University of British Columbia
Medical University of Graz; Hospital de Clinicas de Porto Alegre

Unknown

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Morquio B Disease | GM1 Gangliosidosis Type III

Canada
Allievex Corporation

Completed

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 B

United States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
UniQure Biopharma B.V.
Institut Pasteur; Venn Life Sciences

Completed

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Sanfilippo Syndrome B

France
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
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
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
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

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

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