Search clinical trials for: Mucopolysaccharidosis IV type A

Total 491987 results

Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
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
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
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
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
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
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
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
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
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
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
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
Birmingham Women's and Children's NHS Foundation...
BioMarin Pharmaceutical

Withdrawn

Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa

Mucopolysaccharidosis IV | Morquio A Disease

United Kingdom
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

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
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

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
Emory University
BioMarin Pharmaceutical

Terminated

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

Mucopolysaccharidosis Type IVA

United States
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
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
BioMarin Pharmaceutical

Approved for marketing

BMN 110 US Expanded Access Program

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United States, Puerto Rico
Ohio State University Comprehensive Cancer Center

Not yet recruiting

Ketogenic Dietary Intervention to Improve Response to Immunotherapy in Patients With Metastatic Melanoma and Metastatic Kidney Cancer

Metastatic Renal Cell Carcinoma | Metastatic Cutaneous Melanoma | Stage IV Renal Cell Cancer AJCC v8 | Clinical Stage IV Cutaneous Melanoma AJCC v8

United States
Ivy Brain Tumor Center
GlaxoSmithKline

Not yet recruiting

A Study Comparing Niraparib With Temozolomide in Adult Participants With Newly-diagnosed, MGMT Unmethylated Glioblastoma

Glioblastoma | Brain Tumor | GBM | Brain Neoplasms, Adult, Malignant
Mayo Clinic

Not yet recruiting

Accuracy of 18F-rhPSMA-7.3 PET/ MRI for Prediction of Lymph Node Metastasis in Localized High-Risk Prostate Cancer

Localized Prostate Carcinoma | Stage I Prostate Cancer AJCC v8 | Stage II Prostate Cancer AJCC v8 | Stage III Prostate Cancer AJCC v8 | Stage IVA Prostate Cancer AJCC v8 | Oligometastatic Prostate Carcinoma

United States
Regeneron Pharmaceuticals

Not yet recruiting

Study of Cemiplimab Alone or in Combination With Fianlimab and/or Other Experimental Agents in Adult Participants With Peri-operative Stage III/IV Cutaneous Squamous Cell Carcinoma (CSCC)

Cutaneous Squamous Cell Carcinoma
Mayo Clinic
National Cancer Institute (NCI)

Not yet recruiting

Photoradiation With Verteporfin to Facilitate Immunologic Activity of Pembrolizumab in Unresectable, Locally Advance or Metastatic Pancreatic Cancer

Stage II Pancreatic Cancer AJCC v8 | Stage III Pancreatic Cancer AJCC v8 | Stage IV Pancreatic Cancer AJCC v8 | Metastatic Pancreatic Adenocarcinoma | Locally Advanced Pancreatic Adenocarcinoma | Unresectable Pancreatic Adenocarcinoma

United States
French Innovative Leukemia Organisation
Acute Leukemia French Association

Not yet recruiting

OBServatory of Compassionate Use of IVOsidenib in France for Patients With Acute Myeloid Leukemia

AML, Adult

France
Weill Medical College of Cornell University
Curium US LLC

Not yet recruiting

Feasibility of Intraoperative Tracing of Meningioma Using [Cu64]DOTATATE

Meningioma

United States
Tianjin Medical University Cancer Institute and...

Recruiting

Ivonescimab Combined With HAIC for the Treatment of Unresectable Hepatocellular Carcinoma（uHCC）.

Hepatocellular Carcinoma | Hepatocellular Carcinoma Non-resectable

China
National Cancer Institute (NCI)

Not yet recruiting

Testing the Combination of the Anticancer Drugs Trastuzumab Deruxtecan (DS-8201a) and Azenosertib (ZN-c3) in Patients With Stomach or Other Solid Tumors

Locally Advanced Malignant Solid Neoplasm | Unresectable Malignant Solid Neoplasm | Metastatic Malignant Solid Neoplasm | Clinical Stage III Gastric Cancer AJCC v8 | Clinical Stage III Gastroesophageal Junction Adenocarcinoma AJCC v8 | Clinical Stage IV Gastric Cancer AJCC v8 | Clinical Stage... and other conditions
Beijing Tiantan Hospital
West China Hospital; The First Affiliated Hospital of Zhengzhou University; Capital... and other collaborators

Recruiting

Evaluate the Effectiveness and Safety of Raman IVD Analyzer in the Molecular Diagnosis of Gliomas During Surgery

Glioma

China
AbbVie

Recruiting

A Study to Assess Frequency of Undetectable Minimal Residual Disease (uMRD) in Adult Participants With Chronic Lymphocytic Leukemia (CLL) Receiving Oral Venetoclax Tablets ± Intravenously (IV) Infused Rituximab in Routine Clinical Practice in Japan

Chronic Lymphocytic Leukemia

Japan
Mayo Clinic

Recruiting

Oropharynx (OPX) Biomarker Trial

Oropharyngeal Squamous Cell Carcinoma | Recurrent Oropharyngeal Squamous Cell Carcinoma | Clinical Stage III HPV-Mediated (p16-Positive) Oropharyngeal Carcinoma AJCC v8 | Clinical Stage IV HPV-Mediated (p16-Positive) Oropharyngeal Carcinoma AJCC v8 | Stage III Oropharyngeal (p16-Negative)... and other conditions

United States
Zhejiang Cancer Hospital

Recruiting

A Randomized, Multicenter Phase II Basket Study of Hypofractionated Radiotherapy/Stereotactic Body Radiotherapy Followed by Immunotherapy-Based Systemic Therapy +/- L. Rhamnosus M9 for the First-Line Treatment of Advanced Digestive System Malignancies.

Gastric Cancer | Colorectal Cancer | Pancreatic Adenocarcinoma | Biliary Tract Cancer | Gastroesophageal Junction Adenocarcinoma | Liver Cancer Stage IV

China
Veerle Snijders
Croatian Institute of Public Health; Andrija Štampar School of Public Health

Recruiting

Using Behavioural and Cultural Insights to Increase Colorectal Cancer Home-testing in Croatia

Colorectal Cancer | Behavior Change

Croatia
City of Hope Medical Center

Recruiting

Stomach Cancer Exosome-based Detection (DESTINEX)

Gastric Cancer | Gastric Adenocarcinoma | Gastric Cancer Stage IV | Gastric Neoplasm | Gastric Cancer Metastatic to Lung | Gastric Cancer Stage | Gastric Cancer Metastatic to Liver | Gastric Cancer Stage III | Gastric Cancer Stage II | Gastric Lesion | Gastric Cancer in Situ | Gastric Cancer Stage IIIB | Gastric... and other conditions

United States, Japan
City of Hope Medical Center

Recruiting

Early Detection of Advanced Adenomas and Colorectal Cancer (AACRC)

Colorectal Neoplasms | Colorectal Cancer | Colorectal Disorders | Colorectal Adenocarcinoma | Colorectal Cancer Stage II | Colorectal Cancer Stage III | Colorectal Cancer Stage IV | Colorectal Polyp | Colorectal Neoplasms Malignant | Colorectal Adenomatous Polyp | Colorectal Cancer Stage I | Colorectal Adenoma... and other conditions

United States, Italy, China, Spain, Japan
Clinica Universidad de Navarra, Universidad de...

Not yet recruiting

Advice of Moderate Drinking Pattern Versus Advice on Abstention on Major Disease and Mortality (UNATI)

Depression | Cardiovascular Diseases | Liver Cirrhosis | Infections | Dementia | Type 2 Diabetes | Tuberculosis | All Cause Mortality | Injury Traumatic | Invasive Cancer

Spain
Nationwide Children's Hospital
Pfizer

Not yet recruiting

Lorlatinib for Newly-Diagnosed High-Grade Glioma With ROS or ALK Fusion

Glioblastoma | Glioblastoma Multiforme | High Grade Glioma | Anaplastic Astrocytoma | Diffuse Intrinsic Pontine Glioma | WHO Grade III Glioma | WHO Grade IV Glioma | Infant Type Hemispheric Glioma | Diffuse Midline Glioma, H3K27-altered

United States, Australia, Canada, Germany, Netherlands
Mostafa Bahaa

Not yet recruiting

Doxycycline in Type II Diabetes

Diabetes Mellitus, Type 2
Emory University
National Cancer Institute (NCI)

Not yet recruiting

Personalized Vaccine Immunotherapy in Combination With Checkpoint Inhibitor for Treatment of Triple Negative Breast Cancer

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic... and other conditions

United States
University Health Network, Toronto

Recruiting

Clear Me: Interception Trial to Detect and Clear Molecular Residual Disease in Patients With High-risk Melanoma (ClearMe)

Cutaneous Melanoma | Mucosal Melanoma | Melanoma Stage IV

Canada
National Cancer Institute (NCI)

Not yet recruiting

Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia

B Acute Lymphoblastic Leukemia | Acute Leukemia of Ambiguous Lineage
Thomas Jefferson University

Recruiting

An Interactive Time-Restricted Diet Intervention (txt4fasting) for Reducing Neurocognitive Decline and Improving Survival in Patients With Brain Metastases From Breast or Lung Cancer

Anatomic Stage IV Breast Cancer AJCC v8 | Metastatic Lung Carcinoma | Stage IV Lung Cancer AJCC v8 | Metastatic Malignant Neoplasm in the Brain | Metastatic Breast Carcinoma

United States
The First Hospital of Jilin University

Not yet recruiting

To Explore the Feasibility of Dynamic Changes of TCR Diversity in Peripheral Blood in Monitoring Recurrence and Evaluating Prognosis of Epithelial Ovarian Cancer

Epithelial Ovarian Cancer | Stage IV Ovarian Cancer | Stage III Ovarian Cancer
Damanhour University
Tanta University

Recruiting

Empagliflozin Versus Vildagliptin in CAD Patients With T2DM

Coronary Artery Disease | Type 2 Diabetes

Egypt
Zhejiang Haichang Biotech Co., Ltd.

Not yet recruiting

Phase 2 Study of WGI-0301 in Combination With Sorafenib for Advanced HCC

Advanced Hepatocellular Carcinoma (HCC)
Roswell Park Cancer Institute

Recruiting

Bipolar Androgen Therapy to Restore Sensitivity to Androgen Deprivation Therapy for Patients With Metastatic Castration Resistant Prostate Cancer

Castration-Resistant Prostate Carcinoma | Metastatic Prostate Carcinoma | Stage IVB Prostate Cancer AJCC v8

United States
Attikon Hospital

Recruiting

The Effect of Semaglutide on Cardiovascular Markers and Liver Function

Diabetes Type 2

Greece
Assiut University

Not yet recruiting

Biceps Tenodesis Alone Versus Biceps Tenodesis and Labrum Repair in Superior Labrum Anteroposterior (SLAP) Lesion Type IV

SLAP Lesion

Search clinical trials for: Mucopolysaccharidosis IV type A

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Italy

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations