Clinical Trials on MPS IV A

Total 8266 results

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
Sigilon Therapeutics, Inc.

Withdrawn

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

MPS I | MPS1-S | MPS1-HS

Brazil, 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
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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
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
Nemours Children's Clinic
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States
BioMarin Pharmaceutical

Terminated

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

France, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy
University of Leipzig
Deutsche Krebshilfe e.V., Bonn (Germany)

Recruiting

Study Investigating Patient-Reported Outcomes in Lower-risk MDS Patients (PRO-RED)

MDS | MDS/MPN

Germany, Austria
University Hospital, Geneva
European Society for Blood and Marrow Transplantation

Recruiting

Polymorphisms and Busulfan Pharmacokinetic Study

Children Who Receive a Stem Cell Transplantation With Busulfan IV

Canada, France, Switzerland
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
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
Universitaire Ziekenhuizen KU Leuven

Completed

SecurAcath Versus Statlock for PICC Securement (SecurAstaP)

Patients With Any Condition Who Need a PICC for IV Therapy

Belgium
Children's Hospital of Eastern Ontario

Completed

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

MPS I | MPS IV

Canada
Michael Savona
Incyte Corporation; Astex Pharmaceuticals, Inc.; Theradex

Recruiting

Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO) (ABNL-MARRO)

MDS/MPN

United States
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

Approved for marketing

BMN 110 US Expanded Access Program

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United States, Puerto Rico
Peking University People's Hospital

Recruiting

Assessment of Measurable Residual Disease in Allo-HSCT Using Digital Polymerase Chain Reaction

Acute Leukemia | MDS | CML | MDS/MPN

China
The First Affiliated Hospital with Nanjing Medical...
Jiangbin Hospital Affiliated to Jiangsu University; Nanjing Second Hospital; Jiangning...

Recruiting

Clinical Study of Azacitidine Combined With Ruxolitinib in the Treatment of Higher-risk MDS/MPN

MDS/MPN

China
DKMS gemeinnützige GmbH

Recruiting

GvHD Prophylaxis in Unrelated Donor HCT: Randomized Trial Comparing PTCY Versus ATG (GRAPPA)

AML | MDS | Peripheral Blood Stem Cell Transplantation | Graft Vs Host Disease | CMML | MDS/MPN

Germany
BioMarin Pharmaceutical

Terminated

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United States, United Kingdom, Canada, Germany
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
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
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
Ohio State University Comprehensive Cancer Center

Completed

2 Phase Comparison of Pre-operative CT and PET Images for Metastatic Colorectal Cancer

Recurrent Colon Cancer | Recurrent Rectal Cancer | Stage IV A Bladder Cancer | Stage IV A Rectal Cancer | Stage IV B Colon Cancer | Stage IV B Rectal Cancer

United States
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
Benjamin Tomlinson

Recruiting

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Myelodysplastic Syndromes | MDS/MPN Crossover Syndromes

United States
University of California, San Francisco
Duke University

Recruiting

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

Wolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VII

United States
University of Pittsburgh

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions

United States
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Terminated

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Spain, Australia
GOIZET
BioMarin Pharmaceutical

Recruiting

Natural History of Atypical Morquio A Disease

Mucopolysaccharidosis IV A

France
Gwynn Long, M.D.
ADC Therapeutics S.A.

Terminated

ADCT-301 in Patients With R/R AML, MDS, or MDS/MPN

Acute Myeloid Leukemia (AML) | Myelodysplastic Syndrome (MDS) | Myeloproliferative Neoplasm (MDS/MPN)

United States
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Active, not recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Australia, Spain
Oregon Health and Science University
University of Utah; BioMarin Pharmaceutical

Completed

Gait Analysis in MPS IVA

MPS IVA | Morquio Syndrome

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
Amal Therapeutics
Boehringer Ingelheim

Active, not recruiting

Phase 1b Study to Evaluate ATP128, VSV-GP128 and BI 754091, in Patients With Stage IV Colorectal Cancer (KISIMA-01)

Metastatic Colorectal Cancer | Colorectal Cancer | Liver Metastasis Colon Cancer | Stage IV Colon Cancer | Stage IV Rectal Cancer | MSS

United States, Belgium, Switzerland, Germany
Shandong New Time Pharmaceutical Co., LTD

Completed

A Phase IV Study of Huazhi Rougan Granule

A Phase IV Trial to Investigate the Efficacy and Safety of Huazhi Rougan Granule in the Treatment of Non-alcoholic Simple Fatty Liver

China
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
Jacqueline Garcia, MD
National Institutes of Health (NIH)

Recruiting

Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN

Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML) | Myelodysplastic Syndrome (MDS) | Hematopoietic Stem Cell Transplant | MDS/Myeloproliferative Neoplasm-unclassifiable (MDS/MPN-unclassifiable)

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
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, Israel, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon, Netherlands
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
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
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
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
Peter Szmuk
Luminex Corporation; Outcomes Research Consortium

Completed

Use of Vein Viewer for Insertion of Peripheral Venous Cannula in Pediatric Patients With Difficult Intravenous Access

IV Cannulation

United States
Stanford University
National Institutes of Health (NIH)

Completed

Non-Myeloablative Allogeneic Transplant for Myelodysplastic Syndromes and Myeloproliferative Disorders

Myelodysplastic Syndromes | Myeloproliferative Disorders | Myelodysplastic Syndromes (MDS) | Blood and Marrow Transplant (BMT) | Myeloproliferative Disorders (MPD)

United States
Hadassah Medical Organization
Ministry of Health, Israel

Completed

Evaluation of a Health Promoting Community Center Network (HPCCN) Innovation. (HPCCN)

A Community Center as a Health Promoting Setting

Israel
National Taiwan University Hospital

Completed

Compatibility Problems and Prescribing Pattern of Injectable Drugs at the Hospital

IV Injection

Clinical Trials on MPS IV A

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