Search clinical trials for: mucopolysaccharidoses

Total 225 results

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
University Hospital, Tours

Completed

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

Mucopolysaccharidoses

France
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
Hasan Kalyoncu University

Completed

The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis

Mucopolysaccharidosis

Turkey
IRCCS San Raffaele
Fondazione Telethon

Active, not recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant (TigetT10_MPSIH)

Mucopolysaccharidosis IH

Italy
REGENXBIO Inc.

Enrolling by invitation

Long-term Follow-Up for RGX-121

Mucopolysaccharidosis II

United States
BioMarin Pharmaceutical

Completed

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

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV A

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

Completed

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

Morquio Disease

Pakistan
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
Emory University
BioMarin Pharmaceutical

Completed

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? (MorqPreg)

MPS - Mucopolysaccharidosis | Morquio Disease

United States
Masonic Cancer Center, University of Minnesota
BioMarin Pharmaceutical

Terminated

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Maroteaux-Lamy Syndrome

United States
Assiut University

Unknown

Evaluation of Cardiac Affections in Patients With Mucopolysaccharidosis (MPS) in Assuit University Children Hospital (AUCH) (MPS)

Mucopolysaccharidoses
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
Bellicum Pharmaceuticals

No longer available

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Hurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage Disorder

United States
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
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

United States, Brazil, Mexico, Portugal
Ultragenyx Pharmaceutical Inc

Completed

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7

United States
Hacettepe University

Completed

Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients

Mucopolysaccharidoses

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
Masonic Cancer Center, University of Minnesota

Completed

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Hurler Syndrome

United States
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
Emory University

Completed

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Sickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditions

United States
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

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

Search clinical trials for: mucopolysaccharidoses

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