Clinical Trials on Mucopolysaccharidosis Type VII in United Kingdom

Total 28363 results

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
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
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
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
University of Manchester
University College, London; Manchester University NHS Foundation Trust; Orchard... and other collaborators

Active, not recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis Type IIIA

United Kingdom
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
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
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
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
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
Shire

Completed

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

Mucopolysaccharidosis (MPS)

United Kingdom, Netherlands
Sanguine Biosciences
Denali Therapeutics Inc.

Recruiting

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

Mucopolysaccharidosis III-A

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
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
Abeona Therapeutics, Inc

Terminated

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

Mucopolysaccharidosis Type 3 B

United States, Germany, France, Spain
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
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

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

Sanfilippo Syndrome Type B

United States, United Kingdom, Germany
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
Inventiva Pharma

Completed

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

Mucopolysaccharidosis VI

United Kingdom, France, Germany, Portugal
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
REGENXBIO Inc.

Completed

Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis II

Brazil, United States, United Kingdom
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Enrolling by invitation

A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Mucopolysaccharidosis III-A

United States, Australia, Spain
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
Denali Therapeutics Inc.

Recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Mucopolysaccharidosis Type IIIA

United States
Manchester University NHS Foundation Trust

Completed

Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II (COHERE)

Mucopolysaccharidosis Type II

United Kingdom
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
Masonic Cancer Center, University of Minnesota

Recruiting

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Mucopolysaccharidosis Type I | Hematopoietic Cell Transplantation

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
REGENXBIO Inc.

Active, not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Brazil
REGENXBIO Inc.

Active, not recruiting

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Canada
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
PRA Health Sciences

Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

Sly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7

United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
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
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, 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
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
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
University of California, San Francisco

Recruiting

Registry of Patients Diagnosed With Lysosomal Storage Diseases (LSD Registry)

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher Disease

United States
Lundquist Institute for Biomedical Innovation at...
Swedish Orphan Biovitrum; Cure Sanfilippo Foundation

Completed

Open-label Study of Anakinra in MPS III

Mucopolysaccharidosis III

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
Immusoft of CA, Inc.

Recruiting

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Mucopolysaccharidosis IH/S | Mucopolysaccharidosis IS

United States
LYSOGENE

Completed

Natural History Study of Patients With MPS IIIA

Mucopolysaccharidosis IIIA

Germany, Brazil, United Kingdom, France, Netherlands
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
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia
Phoenix Nest
Aparito Ltd.; Vaincres Les Maladies Lysosomales; Association Sanfilippo Sud

Not yet recruiting

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC (MPSIIIC)

Sanfilippo Syndrome Type C

United States, France
University of Manchester
Manchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaborators

Recruiting

Autologous Ex-vivo Gene Modified HSCT in MPSII

Mucopolysaccharidosis II

United Kingdom
BioMarin Pharmaceutical

Completed

Study of rhASB in Patients With Mucopolysaccharidosis VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Mucopolysaccharidosis VI

United States

Clinical Trials on Mucopolysaccharidosis Type VII in United Kingdom

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