Clinical Trials on Mucopolysaccharidosis in United States

Total 161 results

Immusoft of CA, Inc.

Recruiting

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

Mucopolysaccharidosis IH/S | Mucopolysaccharidosis IS

United States
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
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
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
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
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
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
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
Abeona Therapeutics, Inc

Terminated

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

Mucopolysaccharidosis Type 3 B

United States, Germany, France, Spain
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
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
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
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
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
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
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
BioMarin Pharmaceutical

Completed

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Mucopolysaccharidosis 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
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
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
Emory University
BioMarin Pharmaceutical

Terminated

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

Mucopolysaccharidosis Type IVA

United States
Denali Therapeutics Inc.

Recruiting

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

Mucopolysaccharidosis Type IIIA

United States
Masonic Cancer Center, University of Minnesota

Completed

Intrathecal Enzyme Replacement for Hurler Syndrome

Hurler Syndrome

United States
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
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
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

An Extension Study of JR-171-101 Study in Patients With MPS I

Mucopolysaccharidosis I

United States, Brazil, Japan
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

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
REGENXBIO Inc.

Active, not recruiting

RGX-111 Gene Therapy in Patients With MPS I

Hurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)

United States, Brazil, Israel
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
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Mucopolysaccharidosis I

United States, Brazil, Japan
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
Shire

Completed

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

Mucopolysaccharidosis (MPS)

United Kingdom, Netherlands
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

MPS I

United States
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

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
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
REGENXBIO Inc.

Enrolling by invitation

Long Term Follow-Up for RGX-111

Mucopolysaccharidosis I

United States, Brazil
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
National MPS Society
MediResource Inc.

Terminated

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidoses | Mucopolysaccharidosis VI | Mucopolysaccharidosis IV

United States
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
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
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant w/Laronidase for Hurler

Mucopolysaccharidosis I | Hurler Syndrome

United States
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
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
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

Mucopolysaccharidosis I

United States
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
Lundquist Institute for Biomedical Innovation at...

Not yet recruiting

Study of Cannabidiol in Sanfilippo Syndrome

Mucopolysaccharidosis III | Sanfilippo Syndrome

United States

Clinical Trials on Mucopolysaccharidosis in United States

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